The scientists and clinician-researchers advancing each condition — ranked by published research, with a link to their work. A positive-sum map of who knows a disease best, so patients, orgs and rural clinicians can reach the right expert. It’s your profile — claim it to keep it accurate. Never pay-to-rank.
Division of Neurology
Friedreich Ataxia · through 2026
Pediatric Nephrology
Nephropathic Cystinosis · through 2026
Department of Pediatric Nephrology
Nephropathic Cystinosis · through 2026
Bruce Lefroy Centre for Genetic Health Research
Friedreich Ataxia · through 2026
Division of Nephrology
Nephropathic Cystinosis · through 2026
Department of Pediatric Nephrology
Cystinosis · through 2026
Department of Dietetics
Phenylketonuria · through 2026
Bruce Lefroy Centre for Genetic Health Research
Friedreich Ataxia · through 2026
Division of Endocrinology and Metabolism
Homozygous Familial Hypercholesterolemia · through 2026
Ophthalmology Unit
Vernal Keratoconjunctivitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Oregon Health & Science University, Portland, OR
CTX · through 2025
Department of Medicine
Homozygous Familial Hypercholesterolemia · through 2026
Department of Paediatrics
Homozygous Familial Hypercholesterolemia · through 2026
Pediatric Nephrology
Nephropathic Cystinosis · through 2026
Pediatric Nephrology
Cystinosis · through 2026
Izmir Institute of Technology
Tay-Sachs Disease · through 2026
Department of Neurology
Friedreich Ataxia · through 2026
Plastic Surgery
Neurotrophic Keratopathy · through 2026
Department of Pediatric Kidney
Nephropathic Cystinosis · through 2026
Department of Neurology
Wilson Disease · through 2026
Department of Neurology and Fixel Institute for Neurological Disorders
Friedreich Ataxia · through 2026
Department of Neurology, Canisius Wilhelmina Hospital, Nijmegen, The Netherlands
CTX · through 2024
Department of Medicine, Surgery and Neurosciences, Medical School, University of Siena and
CTX · through 2021
Department of Medicine
Homozygous Familial Hypercholesterolemia · through 2026
Section of Pharmacology
Homocystinuria · through 2026
Department of Pediatrics
Nephropathic Cystinosis · through 2024
Department of Internal Medicine
Nephropathic Cystinosis · through 2023
Division of Nephrology
Cystinosis · through 2026
Department of Dietetics
Phenylketonuria · through 2026
Department of Dietetics
Phenylketonuria · through 2026
Department of Neurology
Friedreich Ataxia · through 2026
Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia · through 2026
Department of Anesthesiology
Postherpetic Neuralgia · through 2026
Centre de Référence Des Maladies Rénales Rares
Nephropathic Cystinosis · through 2026
Laboratory of Nephrology
Nephropathic Cystinosis · through 2025
Laboratory of Nephrology
Nephropathic Cystinosis · through 2024
Department of Clinical and Chemical Pathology
Nephropathic Cystinosis · through 2024
National Clinical Research Center for Digestive Diseases and Xijing Hospital of
Primary Biliary Cholangitis · through 2026
Department of Family Medicine
Central Precocious Puberty · through 2026
Department of Dietetics
Phenylketonuria · through 2026
Department of Dietetics
Phenylketonuria · through 2026
Scott-Ritchey Research Center
Tay-Sachs Disease · through 2026
Department of Neurology
Friedreich Ataxia · through 2026
Department of Neurology
Friedreich Ataxia · through 2026
UO Clinical Neurology and Neurometabolic Diseases, AOU Senese, Siena, Italy
CTX · through 2021
Oregon Health & Science University, Portland, OR
CTX · through 2025
PreventionGenetics, Exact Sciences, Marshfield, Wisconsin; University of Wisconsin School
CTX · through 2024
Vanderbilt University Medical Center
Hypophosphatasia · through 2026
Osteology/Clinical Trial Unit
Hypophosphatasia · through 2026
Shanxi Medical University
Postherpetic Neuralgia · through 2026
Shunde Hospital of Traditional Chinese Medicine
Postherpetic Neuralgia · through 2026
John Walton Muscular Dystrophy Research Centre
Duchenne Muscular Dystrophy · through 2026
Department of Ophthalmology and Vision Science
Neurotrophic Keratopathy · through 2026
Department of Gastroenterology
Primary Biliary Cholangitis · through 2026
Department of Endocrinology
Central Precocious Puberty · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Neurosurgery
Glioma · through 2026
Department of Endocrinology and Metabolism
Wilson Disease · through 2026
Department of Genetic and Cellular Medicine
Tay-Sachs Disease · through 2026
Children's Mercy Kansas City
Hypophosphatasia · through 2026
Centre de référence des microangiopathies thrombotiques
Thrombotic Thrombocytopenic Purpura · through 2026
Department of Pain Treatment
Postherpetic Neuralgia · through 2026
The Fourth Affiliated Hospital
Postherpetic Neuralgia · through 2026
Nephrology and Transplantation Department
Nephropathic Cystinosis · through 2026
Laboratory of Nephrology
Nephropathic Cystinosis · through 2025
Department of Development & Regeneration
Nephropathic Cystinosis · through 2024
Department of Pediatric Kidney
Cystinosis · through 2026
Division of Metabolic Diseases and Hepatology
Cystinosis · through 2026
Department of Public Health
Fabry Disease · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Biomedical Engineering
Pulmonary Tuberculosis · through 2026
Department of Hematology
Autoimmune Hemolytic Anemia · through 2026
Department of Digestive Diseases
Short Bowel Syndrome · through 2026
Department of Pediatric Surgery
Glioma · through 2026
School of Pharmacy and State Key Laboratory of Mechanism and Quality Research in
Rheumatoid arthritis · through 2026
School of Tropical Agriculture and Forestry
Ulcerative colitis · through 2026
Department of Thoracic Surgery
Ulcerative colitis · through 2026
Faculty of Medicine and Health
Myasthenia Gravis · through 2026
Department of Internal Medicine
Wilson Disease · through 2026
Department of Genetic and Cellular Medicine
Tay-Sachs Disease · through 2026
German Center for Neurodegenerative Diseases
Friedreich Ataxia · through 2026
Department of Medicine, Surgery and Neurosciences, Medical School, University of Siena, Si
CTX · through 2021
Swansea University Medical School, ILS1, Swansea, Wales, United Kingdom. Electronic addres
CTX · through 2021
Swansea University Medical School, ILS1, Swansea, Wales, United Kingdom
CTX · through 2021
Department of Internal Medicine Laboratory
Homozygous Familial Hypercholesterolemia · through 2026
Department of Pediatrics
Homozygous Familial Hypercholesterolemia · through 2026
Department of Medicine
Homozygous Familial Hypercholesterolemia · through 2026
FIRMO Foundation
Hypophosphatasia · through 2026
Department of Pediatrics
Hypophosphatasia · through 2026
Department of Pediatrics and Adolescent Medicine
Hypophosphatasia · through 2026
School of Public Health; Beijing Key Laboratory of Environment and Aging
Active Tuberculosis · through 2026
Huadong Hospital
Postherpetic Neuralgia · through 2026
Department of Pediatrics
Nephropathic Cystinosis · through 2026
Laboratory of Nephrology
Nephropathic Cystinosis · through 2025
Department of Pediatric Nephrology
Cystinosis · through 2026
School of Chinese Medicine
Primary Biliary Cholangitis · through 2026
Neurology Unit
Fabry Disease · through 2026
Department of Nursing
Interstitial Cystitis · through 2026
Department of Neurosurgery
Glioma · through 2027
Henan-Macquarie Uni Joint Centre for Biomedical Innovation
Glioma · through 2026
Waldman Department of Dermatology
Psoriasis · through 2026
Department of Neurology
Myasthenia Gravis · through 2026
Department of Infectious Diseases and Liver Disease
Wilson Disease · through 2026
Department of Infectious Diseases and Liver Disease
Wilson Disease · through 2026
Department of Hepatology and Gastroenterology
Wilson Disease · through 2026
Division of Metabolic Diseases
Phenylketonuria · through 2026
Clinical Data Science GmbH
Friedreich Ataxia · through 2026
Departament Ciències Mèdiques Bàsiques
Friedreich Ataxia · through 2026
Departament Ciències Mèdiques Bàsiques
Friedreich Ataxia · through 2026
Departament Ciències Mèdiques Bàsiques
Friedreich Ataxia · through 2026
From the Department of Neurology (B.M.L.S.), Catharina Hospital, Eindhoven; Department of
CTX · through 2022
Pharmacy and Clinical Pharmacology, Amsterdam UMC Locatie AMC, Amsterdam, Netherlands
CTX · through 2026
Department of Clinical Chemistry and Pediatrics, Laboratory Genetic Metabolic Diseases, Em
CTX · through 2026
Department of Neurology, National Hospital Organization Yamagata National Hospital, Japan
CTX · through 2025
Regeneron Pharmaceuticals
Homozygous Familial Hypercholesterolemia · through 2026
Division of Biosciences
Hypophosphatasia · through 2026
University College London Hospital NHS Trust
Thrombotic Thrombocytopenic Purpura · through 2026
The Center for Reference Material and Standardization
Postherpetic Neuralgia · through 2026
The Third Affiliated Hospital of Southern Medical University
Postherpetic Neuralgia · through 2026
The Fourth Affiliated Hospital
Postherpetic Neuralgia · through 2026
Departments of Ophthalmology
Neurotrophic Keratopathy · through 2026
Eye Clinic
Neurotrophic Keratopathy · through 2026
Massachusetts Eye and Ear
Neurotrophic Keratopathy · through 2026
Department of Pediatric Nephrology
Nephropathic Cystinosis · through 2026
Section on Human Biochemical Genetics
Nephropathic Cystinosis · through 2026
Paediatric Nephrology Department
Nephropathic Cystinosis · through 2024
Department of Pediatric Nephrology
Nephropathic Cystinosis · through 2024
Division of Metabolic Diseases and Hepatology
Cystinosis · through 2026
Nephrology Research Unit
Cystinosis · through 2026
National Clinical Research Center for Digestive Diseases and Xijing Hospital of
Primary Biliary Cholangitis · through 2026
Department of Infectious Disease and Liver Disease
Primary Biliary Cholangitis · through 2026
Department of Endocrinology
Central Precocious Puberty · through 2026
Gaucher Unit
Gaucher Disease · through 2026
Murdoch Children's Research Institute
Neurofibromatosis, Type 1 · through 2026
Centre for Molecular Medicine and Therapeutics
Huntington Disease · through 2026
Department of Dermatology
Psoriasis · through 2026
Department of Neurology
Wilson Disease · through 2026
Laboratory of Molecular Biology
Wilson Disease · through 2026
Department of Internal Medicine
Wilson Disease · through 2026
Department of Cardiovascular Medicine
Marfan Syndrome · through 2026
Metabolic Diseases Unit
Phenylketonuria · through 2026
National Institutes of Health
Tay-Sachs Disease · through 2026
Sorbonne Université
Friedreich Ataxia · through 2026
Department of Neurology
Friedreich Ataxia · through 2026
Department of Neurology
Friedreich Ataxia · through 2026
Division of Neurology
Friedreich Ataxia · through 2026
QIMR Berghofer Medical Research Institute
Friedreich Ataxia · through 2026
Departament Ciències Mèdiques Bàsiques
Friedreich Ataxia · through 2026
Department of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC Universi
CTX · through 2023
Department of Laboratory Medicine, Division of Clinical Chemistry, Karolinska University H
CTX · through 2020
Division of Neurology, Department of Clinical Medicine, Universidade Federal do Ceará, For
CTX · through 2026
Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA
CTX · through 2026
Department of Laboratory Medicine, Translational Metabolic Laboratory, Radboud University
CTX · through 2023
Kanazawa University Graduate School of Medical Sciences
Homozygous Familial Hypercholesterolemia · through 2026
Cardiovascular Center
Homozygous Familial Hypercholesterolemia · through 2026
Center for the Study of Atherosclerosis
Homozygous Familial Hypercholesterolemia · through 2026
Centre for Heart Lung Innovation
Homozygous Familial Hypercholesterolemia · through 2026
Department of Vascular Medicine
Homozygous Familial Hypercholesterolemia · through 2026
Department of Internal Medicine
Homozygous Familial Hypercholesterolemia · through 2026
Department of Medicine
Homozygous Familial Hypercholesterolemia · through 2026
Department of Translational and Precision Medicine
Homozygous Familial Hypercholesterolemia · through 2026
Department of Vascular Medicine
Homozygous Familial Hypercholesterolemia · through 2026
Metabolism and Lipids Program
Homozygous Familial Hypercholesterolemia · through 2026
Academic Research Organization
Homozygous Familial Hypercholesterolemia · through 2026
Duke University Medical Center
Hypophosphatasia · through 2026
Department of Osteology and Biomechanics
Hypophosphatasia · through 2026
Sanford Children's Health Research Center
Hypophosphatasia · through 2026
Section of Pharmacology
Homocystinuria · through 2026
Department of Pediatrics and Inherited Metabolic Disorders
Homocystinuria · through 2025
The University of Kansas Medical Center
Thrombotic Thrombocytopenic Purpura · through 2026
The Third Affiliated Hospital of Southern Medical University
Postherpetic Neuralgia · through 2026
The Third Affiliated Hospital of Southern Medical University
Postherpetic Neuralgia · through 2026
Department of Epidemiology and Health Statistics
Postherpetic Neuralgia · through 2026
Cincinnati Children's Hospital Medical Center
Duchenne Muscular Dystrophy · through 2026
Department of Human Genetics
Duchenne Muscular Dystrophy · through 2026
Department of Neurology
Duchenne Muscular Dystrophy · through 2026
Department of Neuroscience
Neurotrophic Keratopathy · through 2026
Department of Ophthalmology
Vernal Keratoconjunctivitis · through 2026
Nephrology and Transplantation Department
Cystinosis · through 2026
University of California
Cystinosis · through 2026
Division of Pediatric Nephrology
Cystinosis · through 2025
Neuroscience Research Institute and Department of Neurobiology
Amyotrophic Lateral Sclerosis · through 2026
Shenzhen Institute of Pharmacovigilance and Risk Management
Primary Biliary Cholangitis · through 2026
Division of Gastroenterology and Hepatology
Primary Biliary Cholangitis · through 2026
Department of Rheumatology and Clinical Immunology
Primary Biliary Cholangitis · through 2026
Department of Infectious Disease and Liver Disease
Primary Biliary Cholangitis · through 2026
Department of Pediatric Endocrinology
Central Precocious Puberty · through 2026
Jiangxi Provincial Children's Hospital; JXHC Key Laboratory of Precise Preventio
Central Precocious Puberty · through 2026
Department of Traditional Chinese Medicine
Central Precocious Puberty · through 2026
Division of Metabolic Diseases
Fabry Disease · through 2026
Lysosomal Disorders Unit
Fabry Disease · through 2026
Division of Cardiology
Fabry Disease · through 2026
Department of Internal Medicine
Gaucher Disease · through 2026
Gaucher Unit
Gaucher Disease · through 2026
Department of Neurology
Spinal Muscular Atrophy · through 2026
Centro Clinico Nemo
Spinal Muscular Atrophy · through 2026
Department of Urology
Interstitial Cystitis · through 2026
STD&HIV/AIDS Prevention and Control Department
Pulmonary Tuberculosis · through 2026
Department of Hematology
Autoimmune Hemolytic Anemia · through 2026
Peking University People's Hospital
Autoimmune Hemolytic Anemia · through 2026
Acadia Pharmaceuticals Inc
Rett Syndrome · through 2026
Division of Neurology
Rett Syndrome · through 2026
Department of Pediatrics
Rett Syndrome · through 2026
State Key Laboratory of Cancer Biology
Glioma · through 2026
High Magnetic Field Laboratory
Rheumatoid arthritis · through 2026
Department of Dermatology
Psoriasis · through 2026
Jiangsu Key Laboratory For Functional Substance of Chinese Medicine
Ulcerative colitis · through 2026
Department of Endocrinology and Metabolism
Wilson Disease · through 2026
Department of Neurology
Wilson Disease · through 2026
Department of Neurology
Wilson Disease · through 2026
National Reference Center for Wilson's Disease and Other Copper-Related Rare Dis
Wilson Disease · through 2026
Department of Medicine
Ehlers-Danlos Syndrome · through 2026
Division of Inherited Metabolic Diseases
Phenylketonuria · through 2026
Department of Medicine
Tay-Sachs Disease · through 2025
Sorbonne Université
Friedreich Ataxia · through 2026
Pharmacy and Clinical Pharmacology, Amsterdam UMC Locatie AMC, Amsterdam, Netherlands
CTX · through 2026
Department of Medical, Surgical and Neurological Sciences, University of Siena, Italy
CTX · through 2016
Graduate Program in Medicine, Medical Sciences, Universidade Federal do Rio Grande do Sul,
CTX · through 2025
German Center for Neurodegenerative Diseases (DZNE), Tübingen, Germany; Department of Neur
CTX · through 2021
Swansea University Medical School, ILS1 Building, Singleton Park, Swansea SA2 8PP, UK
CTX · through 2019
İstanbul University-Cerrahpaşa, Cerrahpaşa Medical Faculty, Division of Pediatric Nutritio
CTX · through 2026
Epidemiology and Real World Science
Hypophosphatasia · through 2026
Department of Osteology and Biomechanics
Hypophosphatasia · through 2026
IIS La Princesa
Hypophosphatasia · through 2026
University of Manitoba
Hypophosphatasia · through 2026
Key Laboratory of Blood-stasis-toxin Syndrome of Zhejiang Province School of Bas
Active Tuberculosis · through 2026
Key Laboratory of Blood-stasis-toxin Syndrome of Zhejiang Province School of Bas
Active Tuberculosis · through 2026
Department of Respiratory Sciences
Active Tuberculosis · through 2026
Institute for Tuberculosis Control
Active Tuberculosis · through 2026
Department of Chronic Communicable Disease
Active Tuberculosis · through 2026
Department of Pediatric Endocrinology and Genetic Metabolism
Homocystinuria · through 2026
Section of Pharmacology
Homocystinuria · through 2026
Cancer Signaling and Microenvironment Program
Homocystinuria · through 2024
Hebei Provincial People's Hospital
Thrombotic Thrombocytopenic Purpura · through 2026
Hematology Department
Thrombotic Thrombocytopenic Purpura · through 2026
Division of Hematologic Malignancies and Cellular Therapeutics
Thrombotic Thrombocytopenic Purpura · through 2026
Laboratory for Thrombosis Research
Thrombotic Thrombocytopenic Purpura · through 2026
The Third Affiliated Hospital of Southern Medical University
Postherpetic Neuralgia · through 2026
Department of Pain Management
Postherpetic Neuralgia · through 2026
College of Basic Medical Sciences
Postherpetic Neuralgia · through 2026
College of Acupuncture and Massage
Postherpetic Neuralgia · through 2026
Department of Pain Medicine
Postherpetic Neuralgia · through 2026
Children's Hospital of Eastern Ontario Research Institute
Duchenne Muscular Dystrophy · through 2026
Jiangsu Key Laboratory of Tissue Engineering and Neuroregeneration
Duchenne Muscular Dystrophy · through 2026
Key Laboratory of Obstetric and Gynecologic and Pediatric Diseases and Birth Def
Duchenne Muscular Dystrophy · through 2026
Plastic Surgery
Neurotrophic Keratopathy · through 2026
Department of Ophthalmology
Neurotrophic Keratopathy · through 2026
Cornea Service
Neurotrophic Keratopathy · through 2024
Department of Ophthalmology and Visual Science
Vernal Keratoconjunctivitis · through 2026
Società Italiana di Medicina Legale e delle Assicurazioni (SIMLA)
Vernal Keratoconjunctivitis · through 2026
Prof. Brien Holden Eye Research Centre
Vernal Keratoconjunctivitis · through 2026
CODE Eye Care
Vernal Keratoconjunctivitis · through 2026
Department of Ophthalmology
Vernal Keratoconjunctivitis · through 2025
Pediatric Nephrology
Cystinosis · through 2026
Department of Ophthalmology
Cystinosis · through 2026
Heidelberg University
Cystinosis · through 2026
Amalia Children's Hospital
Cystinosis · through 2026
Nephrology Research Unit
Cystinosis · through 2026
Nephrology Research Unit
Cystinosis · through 2026
Paediatric Nephrology Department
Cystinosis · through 2024
Department of Neuromuscular Diseases
Amyotrophic Lateral Sclerosis · through 2026
Department of Colorectal Surgery
Primary Biliary Cholangitis · through 2026
Liver Institute Northwest and Elon S. Floyd College of Medicine
Primary Biliary Cholangitis · through 2026
University of Miami School of Medicine
Primary Biliary Cholangitis · through 2026
The Autoimmune and Rare Liver Disease Programme
Primary Biliary Cholangitis · through 2026
Department of Respiratory and Critical Care Medicine
Primary Biliary Cholangitis · through 2026
Department of Gastroenterology
Primary Biliary Cholangitis · through 2026
Research Center for Clinical Neuroimmunology and Neuroscience
Multiple sclerosis · through 2026
Department of Pediatric Endocrinology
Central Precocious Puberty · through 2026
Ningbo Ninth Hospital Medical Health Group
Central Precocious Puberty · through 2026
Department of Endosecretory Genetic and Metabolic Diseases
Central Precocious Puberty · through 2026
Shenzhen Maternity and Child Healthcare Hospital
Central Precocious Puberty · through 2026
Department of Cardiology
Fabry Disease · through 2026
Division of Metabolic Diseases
Fabry Disease · through 2026
Department of Medicine-DIMED
Fabry Disease · through 2026
Nephrology
Fabry Disease · through 2026
Department of Clinical Medicine and Surgery
Fabry Disease · through 2026
Department of Experimental and Clinical Medicine
Fabry Disease · through 2026
Department of Nephrology
Fabry Disease · through 2026
Department of Pediatrics
Gaucher Disease · through 2026
Section on Molecular Neurogenetics
Gaucher Disease · through 2026
Gaucher Unit
Gaucher Disease · through 2026
Department of Internal Medicine National Taiwan University Hospital Taipei Taiwa
Pulmonary Tuberculosis · through 2026
National Clinical Research Center for Respiratory Disease
Pulmonary Tuberculosis · through 2026
Department of Tuberculosis Control
Pulmonary Tuberculosis · through 2026
Department of Behavioral Sciences and Social Medicine
Neurofibromatosis, Type 1 · through 2026
The Children's Hospital at Westmead
Neurofibromatosis, Type 1 · through 2026
Department of Hematology
Autoimmune Hemolytic Anemia · through 2026
Department of Hematology
Autoimmune Hemolytic Anemia · through 2026
Department of Research and Foreign Affairs
Autoimmune Hemolytic Anemia · through 2026
Peking University People's Hospital
Autoimmune Hemolytic Anemia · through 2026
Université de Paris
Short Bowel Syndrome · through 2026
Anlitiks Inc
Rett Syndrome · through 2026
Department of Molecular and Human Genetics
Rett Syndrome · through 2026
Department of Pediatrics (Neurology)
Rett Syndrome · through 2026
State Key Laboratory of Cancer Biology
Glioma · through 2027
Key Laboratory of Clinical Laboratory Diagnostics (Chinese Ministry of Education
Glioma · through 2026
Department of Clinical Pharmacy
Glioma · through 2026
Henan-Macquarie Uni Joint Centre for Biomedical Innovation
Glioma · through 2026
Institute of Cancer Research
Glioma · through 2026
School of Pharmacy and State Key Laboratory of Mechanism and Quality Research in
Rheumatoid arthritis · through 2026
Department of Health Promotion and Behavioral Sciences
Rheumatoid arthritis · through 2026
Department of Rheumatology
Rheumatoid arthritis · through 2026
Department of Infectious Diseases
Rheumatoid arthritis · through 2026
College of Food Science and Biotechnology
Rheumatoid arthritis · through 2026
Department of Clinical Pharmacology
Rheumatoid arthritis · through 2026
Department of Neurology
Huntington Disease · through 2026
Clinical Research Center (CRC)
Psoriasis · through 2026
Department of Pharmacognosy
Psoriasis · through 2026
Yantai Key Laboratory of Nanomedicine & Advanced Preparations
Ulcerative colitis · through 2026
School of Tropical Agriculture and Forestry
Ulcerative colitis · through 2026
Department of Gastroenterology
Ulcerative colitis · through 2026
Department of Integrative Medicine
Asthma · through 2026
Department of Allergy and Clinical Immunology
Asthma · through 2026
Department of Endocrinology and Metabolism
Wilson Disease · through 2026
The Roger Williams Institute of Liver Studies
Wilson Disease · through 2026
Departments of Medicine and Surgery
Wilson Disease · through 2026
Department of Neurology
Wilson Disease · through 2026
Division of Cardiovascular Medicine
Ehlers-Danlos Syndrome · through 2026
Department of Medicine
Ehlers-Danlos Syndrome · through 2026
Center for Medical Genetics
Ehlers-Danlos Syndrome · through 2026
University Children's Hospital
Phenylketonuria · through 2026
Department of Chemistry
Phenylketonuria · through 2026
Department of Medical and Molecular Genetics
Phenylketonuria · through 2026
PTC Therapeutics
Phenylketonuria · through 2026
Institute for the Study of Inborn Errors of Metabolism
Tay-Sachs Disease · through 2026
Department of Genetic and Cellular Medicine
Tay-Sachs Disease · through 2026
Advanced Therapies Program
Tay-Sachs Disease · through 2024
Platform Medicine for Society, Amsterdam UMC Location University of Amsterdam, Amsterdam,
CTX · through 2026
Swansea University Medical School, ILS1, Swansea, Wales, United Kingdom
CTX · through 2021
Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA
CTX · through 2026
Servicio de Movimientos Anormales, Departamento de Neurología Fleni, Buenos Aires, Argenti
CTX · through 2026
Pharmacy and Clinical Pharmacology, Amsterdam UMC Locatie AMC, Amsterdam, Netherlands n.bo
CTX · through 2026
Service Universitaire de Psychiatrie de l'Enfant et de l'Adolescent, Université de Nantes,
CTX · through 2018
Division of Neurology, Department of Clinical Medicine, Universidade Federal do Ceará, Cea
CTX · through 2025
Inborn Errors of Metabolism, Genetics and Genomic Medicine, UCL Great Ormond Street Instit
CTX · through 2025
Mirum Pharmaceuticals, Inc, Foster City, CA
CTX · through 2025
Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine
CTX · through 2021
Bioinformatics and Data Science
Hypophosphatasia · through 2026
Global Medical Affairs
Hypophosphatasia · through 2026
Paris-Saclay University
Hypophosphatasia · through 2026
Centre for Genomics Research
Hypophosphatasia · through 2026
National Heart and Lung Institute
Cystic Fibrosis · through 2026
Department of Respiratory Medicine
Active Tuberculosis · through 2026
Division of Pulmonary and Critical Care Medicine
Active Tuberculosis · through 2026
Department of Tuberculosis Control and Prevention
Active Tuberculosis · through 2026
Department of Clinical Laboratory
Active Tuberculosis · through 2026
The Baoan People's Hospital of Shenzhen
Active Tuberculosis · through 2026
Department of Chronic Communicable Disease
Active Tuberculosis · through 2026
Department of Pediatric Endocrinology and Genetic Metabolism
Homocystinuria · through 2026
Section of Pharmacology
Homocystinuria · through 2026
Section of Pharmacology
Homocystinuria · through 2026
Department of Pediatric Endocrinology and Genetic Metabolism
Homocystinuria · through 2025
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Thrombotic Thrombocytopenic Purpura · through 2026
Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT)
Thrombotic Thrombocytopenic Purpura · through 2026
Department of Neurology
Postherpetic Neuralgia · through 2026
Department of Acupuncture
Postherpetic Neuralgia · through 2026
Division of Pediatric Cardiology
Duchenne Muscular Dystrophy · through 2026
Child Neurology Unit e Centro Nemo
Duchenne Muscular Dystrophy · through 2026
Eye School of Chengdu University of TCM
Duchenne Muscular Dystrophy · through 2026
Eye School of Chengdu University of TCM
Duchenne Muscular Dystrophy · through 2026
Dubowitz Neuromuscular Centre
Duchenne Muscular Dystrophy · through 2026
Massachusetts Eye and Ear
Neurotrophic Keratopathy · through 2026
University of California
Neurotrophic Keratopathy · through 2026
Shantilal Shanghvi Cornea Institute
Vernal Keratoconjunctivitis · through 2026
Department of Ophthalmology and Vision Sciences
Vernal Keratoconjunctivitis · through 2026
Department of Maternal and Child Area - SC Pediatria Pneumoinfettivologia
Vernal Keratoconjunctivitis · through 2025
Department of Neuroscience
Vernal Keratoconjunctivitis · through 2025
CJ Shah Cornea Services/Dr G Sitalakshmi Memorial Clinic for Ocular Surface Diso
Vernal Keratoconjunctivitis · through 2025
CJ Shah Cornea Services/Dr G Sitalakshmi Memorial Clinic for Ocular Surface Diso
Vernal Keratoconjunctivitis · through 2025
Ophthalmology Unit
Vernal Keratoconjunctivitis · through 2025
Allergy Unit
Vernal Keratoconjunctivitis · through 2025
Allergy Unit
Vernal Keratoconjunctivitis · through 2025
Bichat Hospital and Foundation A. de Rothschild
Vernal Keratoconjunctivitis · through 2024
Amalia Children's Hospital
Cystinosis · through 2026
Advanced Disease Modelling
Cystinosis · through 2026
Department of Neurology
Amyotrophic Lateral Sclerosis · through 2026
Yong Loo Lin School of Medicine
Primary Biliary Cholangitis · through 2026
Department of Gastroenterology and Hepatology
Primary Biliary Cholangitis · through 2026
Division of Gastroenterology and Liver Unit
Primary Biliary Cholangitis · through 2026
National Clinical Research Center for Digestive Diseases and Xijing Hospital of
Primary Biliary Cholangitis · through 2026
Translational and Clinical Research Institute
Primary Biliary Cholangitis · through 2026
Queensland Brain Institute
Multiple sclerosis · through 2026
Department of Neuropsychiatry
Multiple sclerosis · through 2026
Department of Pediatric Endocrinology
Central Precocious Puberty · through 2026
Department of Endocrinology/Pediatric Endocrinology
Central Precocious Puberty · through 2026
Department of Endocrinology
Central Precocious Puberty · through 2026
Division of Endocrinology and Metabolism
Central Precocious Puberty · through 2026
Department of Endocrinology
Central Precocious Puberty · through 2026
Department of Traditional Chinese Medicine
Central Precocious Puberty · through 2026
CAS Key Laboratory of Tissue Microenvironment and Tumor
Central Precocious Puberty · through 2026
Department of Endocrinology
Central Precocious Puberty · through 2026
Cardiology Unit
Fabry Disease · through 2026
Department of Radiology
Fabry Disease · through 2026
Department of Cardiovascular Sciences-CUORE
Fabry Disease · through 2026
Nephrology
Fabry Disease · through 2026
Department of Internal Medicine I
Fabry Disease · through 2026
Department of Pediatrics
Fabry Disease · through 2026
Department of Medical and Cardiovascular Sciences
Fabry Disease · through 2026
Department of Medical and Surgical Sciences (DIMEC)
Fabry Disease · through 2026
Section of Biochemical Genetics
Gaucher Disease · through 2026
Gaucher Unit
Gaucher Disease · through 2026
Lysosomal and Rare Disorders Research and Treatment Center
Gaucher Disease · through 2026
Medical Genetics Branch
Gaucher Disease · through 2026
Discipline of Pediatrics and Child Health
Spinal Muscular Atrophy · through 2026
Department of Cellular and Molecular Biology
Spinal Muscular Atrophy · through 2026
Center for Obstetrics and Gynecology
Spinal Muscular Atrophy · through 2026
Center for Experimental Neurotherapeutics
Spinal Muscular Atrophy · through 2026
Department of Urology
Interstitial Cystitis · through 2026
College of Pharmaceutical Science
Interstitial Cystitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Radiology
Pulmonary Tuberculosis · through 2026
Department of ICU
Pulmonary Tuberculosis · through 2026
Department of Radiology
Pulmonary Tuberculosis · through 2026
College of Life Science and Technology
Pulmonary Tuberculosis · through 2026
Department of Occupational Respiratory Diseases
Pulmonary Tuberculosis · through 2026
Department of Behavioral Sciences and Social Medicine
Neurofibromatosis, Type 1 · through 2026
Suzhou Medical College of Soochow University
Neurofibromatosis, Type 1 · through 2026
Department of Neurosurgery
Neurofibromatosis, Type 1 · through 2026
Pediatric Oncology Branch
Neurofibromatosis, Type 1 · through 2026
Children's National Hospital
Neurofibromatosis, Type 1 · through 2026
Department of Pediatrics
Neurofibromatosis, Type 1 · through 2026
Department of Clinical Laboratory
Autoimmune Hemolytic Anemia · through 2026
Department of Hematology
Autoimmune Hemolytic Anemia · through 2026
Blood and Marrow Transplantation Center
Autoimmune Hemolytic Anemia · through 2026
State Key Laboratory of Experimental Hematology
Autoimmune Hemolytic Anemia · through 2026
Ironwood Pharmaceuticals
Short Bowel Syndrome · through 2026
Division of Pediatric Surgery
Short Bowel Syndrome · through 2026
Translational Research Center for Gastrointestinal Disorders (TARGID)
Short Bowel Syndrome · through 2026
F.M. Kirby Neurobiology Center
Rett Syndrome · through 2026
University of Wisconsin-Madison
Rett Syndrome · through 2026
Anlitiks Inc
Rett Syndrome · through 2026
Department of Molecular and Human Genetics
Rett Syndrome · through 2026
Vanderbilt Kennedy Center
Rett Syndrome · through 2026
State Key Laboratory of Cancer Biology
Glioma · through 2026
Department of Medical Oncology
Glioma · through 2026
Department of Neurosurgery
Glioma · through 2026
Center for Cancer & Blood Disorders
Glioma · through 2026
Department of Pharmacy
Glioma · through 2026
Emergency Department
High blood pressure · through 2026
Department of Neurology
High blood pressure · through 2026
Department of Rheumatology and Immunology
Rheumatoid arthritis · through 2026
School of Public Health
Rheumatoid arthritis · through 2026
Key Laboratory of Environment and Health
Rheumatoid arthritis · through 2026
Trauma Treatment Center
Rheumatoid arthritis · through 2026
BGI Research
Rheumatoid arthritis · through 2026
School of Life Sciences
Rheumatoid arthritis · through 2026
Undergraduate of College of Public Health
Rheumatoid arthritis · through 2026
Health Services Research Division
Huntington Disease · through 2026
Department of Human Genetics
Huntington Disease · through 2026
Department of Neurology University of Minnesota Medical School Minneapolis Minne
Epilepsy · through 2026
Department of Neurology
Epilepsy · through 2026
Department of Gastroenterology
Psoriasis · through 2026
Department of Dermatology
Psoriasis · through 2026
Division of Dermatology
Psoriasis · through 2026
The Second Affiliated Hospital of Hunan University of Chinese Medicine
Psoriasis · through 2026
Baoding Key Laboratory of Cancer & Aging
Psoriasis · through 2026
Departments of Laboratory Medicine
Psoriasis · through 2026
Yunnan Key Laboratory of Dai and Yi Medicines
Ulcerative colitis · through 2026
Department of Gastroenterology
Ulcerative colitis · through 2026
Institute of Basic Medical Sciences & School of Basic Medicine
Asthma · through 2026
Shanghai Institute of Infectious Diseases and Biosecurity
Asthma · through 2026
Department of Respiratory Medicine
Asthma · through 2026
Department of Respiratory Medicine
Asthma · through 2026
Department of Dermatology
Asthma · through 2026
Department of Pediatrics
Asthma · through 2026
Department of Cardiothoracic Surgery
Myasthenia Gravis · through 2026
Department of Neurology
Myasthenia Gravis · through 2026
Department of Neurology
Myasthenia Gravis · through 2026
Departments of Biostatistics
Myasthenia Gravis · through 2026
Department of Neurology
Myasthenia Gravis · through 2026
Charité-Universitätsmedizin Berlin
Myasthenia Gravis · through 2026
Department of Infectious Diseases and Liver Disease
Wilson Disease · through 2026
First Department of Encephalopathy
Wilson Disease · through 2026
Liver Unit
Wilson Disease · through 2026
Center for Medical Genetics
Marfan Syndrome · through 2026
Department of Cardiology
Marfan Syndrome · through 2026
Division of Vascular and Endovascular Surgery
Ehlers-Danlos Syndrome · through 2026
Department of Cardiovascular Medicine
Ehlers-Danlos Syndrome · through 2026
Department of General Internal Medicine
Ehlers-Danlos Syndrome · through 2026
Center for Medical Genetics
Ehlers-Danlos Syndrome · through 2026
North Bristol Trust
Phenylketonuria · through 2026
Nutrition and Metabolism
Phenylketonuria · through 2026
CHRU et Université de Tours
Phenylketonuria · through 2026
Birmingham Children's Hospital
Phenylketonuria · through 2026
PTC Therapeutics
Phenylketonuria · through 2026
Institute for the Study of Inborn Errors of Metabolism
Tay-Sachs Disease · through 2026
Department of Pediatrics
Tay-Sachs Disease · through 2026
Department of Molecular Biology and Genetics
Tay-Sachs Disease · through 2026
Foundation for Research in Genetics and Endocrinology
Tay-Sachs Disease · through 2026
RICN Hospital
Tay-Sachs Disease · through 2026
RICN Hospital
Tay-Sachs Disease · through 2026
Department of Radiology and
Tay-Sachs Disease · through 2025
Department of Biomedical and Molecular Sciences
Tay-Sachs Disease · through 2025
İzmir Institute of Technology
Tay-Sachs Disease · through 2025
Office of the Clinical Director and Medical Genetics Branch
Tay-Sachs Disease · through 2025
Center for Rare Neurological Diseases
Tay-Sachs Disease · through 2025
Department of Clinical Chemistry and Pediatrics, Laboratory Genetic Metabolic Diseases, Em
CTX · through 2025
Division of Neurology, Department of Clinical Medicine, Universidade Federal do Ceará, For
CTX · through 2026
Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO
CTX · through 2025
CNRS-UMR 7241/INSERM U1050, CIRB, Collège de France, UPMC, Paris, France
CTX · through 2018
İstanbul University-Cerrahpaşa, Cerrahpaşa Medical Faculty, Division of Pediatric Nutritio
CTX · through 2026
Metabolic Biochemistry Unit, Department of Biochemistry of Neurometabolic Diseases, Pitié-
CTX · through 2022
MedDay Pharmaceuticals, Institut du Cerveau et de la Moelle épinière, Paris, France
CTX · through 2016
Dipartimento di Medicina Molecolare e Biotecnologie Mediche, Università Degli Studi di Nap
CTX · through 2026
Dipartimento di Medicina Molecolare e Biotecnologie Mediche, Università Degli Studi di Nap
CTX · through 2026
School of Medicine and Surgery, University of Milano-Bicocca, Milan, Italy
CTX · through 2020
Laboratory Research Unit, Experimental Research Center, Hospital de Clínicas de Porto Aleg
CTX · through 2023
Dokuz Eylul University, Medical Faculty, Division of Pediatric Nutrition and Metabolism, I
CTX · through 2026
Division of Neurology, Department of Clinical Medicine, Universidade Federal do Ceará, For
CTX · through 2026
Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine
CTX · through 2020
Neurology Department, Reference Center for Neurological Metabolic and Lysosomal Diseases,
CTX · through 2024
Department of Pharmacy and Clinical Pharmacology, Amsterdam UMC Location University of Ams
CTX · through 2026
İstanbul University-Cerrahpaşa, Cerrahpaşa Medical Faculty, Division of Pediatric Nutritio
CTX · through 2026
Division of Bone and Mineral Diseases
Hypophosphatasia · through 2026
Department of Pediatric Neurology
Hypophosphatasia · through 2026
Department of Microbiology and Immunology
Cystic Fibrosis · through 2026
Department of Gastroenterology
Cystic Fibrosis · through 2026
Department of Pediatric Respiratory Medicine
Cystic Fibrosis · through 2026
Beijing Key Laboratory of Technology and Application for Anti-Infective New Drug
Cystic Fibrosis · through 2026
Department of Respiratory Medicine Bangabandhu Sheikh Mujib Medical University D
Cystic Fibrosis · through 2026
Almazov National Research Medical Center
Active Tuberculosis · through 2026
Almazov National Research Medical Center
Active Tuberculosis · through 2026
Department of Mathematics and Computer Science
Active Tuberculosis · through 2026
Medical Department
Active Tuberculosis · through 2026
Medical Department
Active Tuberculosis · through 2026
Department of Pharmacology
Active Tuberculosis · through 2026
Department of Molecular Biology
Active Tuberculosis · through 2026
Dongguan Sixth People's Hospital
Active Tuberculosis · through 2026
Department of Surgical Intensive Care Unit
Homocystinuria · through 2026
Shenzhen Eye Hospital
Homocystinuria · through 2026
Alexion Pharmaceuticals
Homocystinuria · through 2026
Department of Ophthalmology
Homocystinuria · through 2026
Department of Endocrinology
Homocystinuria · through 2026
Department of Endocrinology
Homocystinuria · through 2026
Anterior Segment Division
Homocystinuria · through 2025
Department of Pediatric Endocrinology and Genetic Metabolism
Homocystinuria · through 2025
Department of Pediatric Endocrinology and Genetic Metabolism
Homocystinuria · through 2025
Department of Pediatric Endocrinology and Genetic Metabolism
Homocystinuria · through 2025
Department of Hematology
Thrombotic Thrombocytopenic Purpura · through 2026
Division of Hematology
Thrombotic Thrombocytopenic Purpura · through 2026
Department of Clinical Pharmacology
Thrombotic Thrombocytopenic Purpura · through 2026
Department of Clinical Pharmacology
Thrombotic Thrombocytopenic Purpura · through 2026
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Thrombotic Thrombocytopenic Purpura · through 2026
Department II of Internal Medicine and Center for Molecular Medicine Cologne
Thrombotic Thrombocytopenic Purpura · through 2026
Second Propedeutic Department of Internal Medicine
Thrombotic Thrombocytopenic Purpura · through 2026
Laboratory for Thrombosis Research
Thrombotic Thrombocytopenic Purpura · through 2026
Shanxi Medical University
Postherpetic Neuralgia · through 2026
Department of Health Sciences
Duchenne Muscular Dystrophy · through 2026
Department of Pharmaceutical Sciences
Duchenne Muscular Dystrophy · through 2026
ReveraGen BioPharma
Duchenne Muscular Dystrophy · through 2026
The John Walton Muscular Dystrophy Research Centre
Duchenne Muscular Dystrophy · through 2026
Davis Health System
Duchenne Muscular Dystrophy · through 2026
Cincinnati Children's Hospital Medical Center
Duchenne Muscular Dystrophy · through 2026
Key Laboratory of Obstetric and Gynecologic and Pediatric Diseases and Birth Def
Duchenne Muscular Dystrophy · through 2026
Department of Biomedical Data Sciences
Duchenne Muscular Dystrophy · through 2026
Chengdu Bio-HT Company Limited
Neurotrophic Keratopathy · through 2026
Department of Ophthalmology
Neurotrophic Keratopathy · through 2026
Department of Ophthalmology
Neurotrophic Keratopathy · through 2026
Massachusetts Eye and Ear
Neurotrophic Keratopathy · through 2026
Department of Cornea and Refractive Surgery
Neurotrophic Keratopathy · through 2026
Department of Cornea and Refractive Surgery
Neurotrophic Keratopathy · through 2026
Department of Ophthalmology
Neurotrophic Keratopathy · through 2026
School of Basic Medicine
Neurotrophic Keratopathy · through 2025
Department of Ophthalmology
Neurotrophic Keratopathy · through 2025
Department of Pediatrics
Vernal Keratoconjunctivitis · through 2026
Department of Ophthalmology and Visual Science
Vernal Keratoconjunctivitis · through 2026
Department of Cornea and Anterior Segment Services
Vernal Keratoconjunctivitis · through 2026
Laboratory of Gene Therapy
Amyotrophic Lateral Sclerosis · through 2026
State Key Laboratory of Genetics and Development of Complex Phenotypes
Amyotrophic Lateral Sclerosis · through 2026
Department of Neurology
Amyotrophic Lateral Sclerosis · through 2026
Department of Convergence Medicine
Amyotrophic Lateral Sclerosis · through 2026
School of Clinical Medicine
Multiple sclerosis · through 2026
Research Center for Clinical Neuroimmunology and Neuroscience
Multiple sclerosis · through 2026
Research Center for Clinical Neuroimmunology and Neuroscience
Multiple sclerosis · through 2026
Tianjin Institute of Immunology
Multiple sclerosis · through 2026
Department of Clinical Neurosciences
Multiple sclerosis · through 2026
Centre d'Esclerosi Múltiple de Catalunya (Cemcat)
Multiple sclerosis · through 2026
University of Genoa
Multiple sclerosis · through 2026
Department of Neurology
Multiple sclerosis · through 2026
Department of Pediatric Endocrinology
Central Precocious Puberty · through 2026
Department of Pediatrics Clinical Medicine
Central Precocious Puberty · through 2026
The First Affiliated Hospital
Gaucher Disease · through 2026
Gaucher Unit
Gaucher Disease · through 2026
Gaucher Unit
Gaucher Disease · through 2026
Gaucher Unit
Gaucher Disease · through 2026
Service d'Hématologie Biologique et service d'Hématologie Clinique Adulte
Gaucher Disease · through 2026
Department of Pharmacology
Gaucher Disease · through 2026
Manchester Foundation Trust
Gaucher Disease · through 2025
Department of Clinical Neuroscience
Gaucher Disease · through 2025
Department of Pediatric Intensive Care Unit
Spinal Muscular Atrophy · through 2026
Department of Neurology
Spinal Muscular Atrophy · through 2026
MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre
Spinal Muscular Atrophy · through 2026
Discipline of Pediatrics and Child Health
Spinal Muscular Atrophy · through 2026
Department of Neurology
Spinal Muscular Atrophy · through 2026
Centro Clinico Nemo
Spinal Muscular Atrophy · through 2026
Horae Gene Therapy Center
Spinal Muscular Atrophy · through 2026
Department of Urology Surgery
Interstitial Cystitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Interstitial Cystitis Medicine
Interstitial Cystitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Arthur Smith Institute for Urology
Interstitial Cystitis · through 2026
Department of Urology
Interstitial Cystitis · through 2026
Department of Nursing
Interstitial Cystitis · through 2026
Department of Radiology
Pulmonary Tuberculosis · through 2026
Department of Community Nursing
Pulmonary Tuberculosis · through 2026
Respiratory Medicine
Pulmonary Tuberculosis · through 2026
Department of Tuberculosis
Pulmonary Tuberculosis · through 2026
Qingchun Hospital
Pulmonary Tuberculosis · through 2026
Department of Pathophysiology
Pulmonary Tuberculosis · through 2026
The First Affiliated Hospital of Shihezi University
Pulmonary Tuberculosis · through 2026
Pediatric Oncology Branch
Neurofibromatosis, Type 1 · through 2026
Neuro-Oncology Division
Neurofibromatosis, Type 1 · through 2026
Department of Pediatric Urology
Neurofibromatosis, Type 1 · through 2026
Department of Psychological and Brain Sciences
Neurofibromatosis, Type 1 · through 2026
University of Manchester & Manchester University Foundation NHS Trust
Neurofibromatosis, Type 1 · through 2026
Center for Autism Services
Neurofibromatosis, Type 1 · through 2026
Department of Neurosurgery
Neurofibromatosis, Type 1 · through 2026
Department of Rheumatology and Clinical Immunology
Autoimmune Hemolytic Anemia · through 2026
School of Public Health
Autoimmune Hemolytic Anemia · through 2026
Internal Medicine
Autoimmune Hemolytic Anemia · through 2026
State Key Laboratory of Common Mechanism Research for Major Diseases
Autoimmune Hemolytic Anemia · through 2026
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Autoimmune Hemolytic Anemia · through 2026
State Key Laboratory of Experimental Hematology
Autoimmune Hemolytic Anemia · through 2026
Department of Pediatric Surgery
Short Bowel Syndrome · through 2026
Division of Pediatric Gastroenterology
Short Bowel Syndrome · through 2026
Division of Pediatric Gastroenterology
Short Bowel Syndrome · through 2026
Division of Pediatric Gastroenterology
Short Bowel Syndrome · through 2026
Intestinal Failure Unit
Short Bowel Syndrome · through 2026
Division of Gastroenterology
Short Bowel Syndrome · through 2026
Ironwood Pharmaceutical
Short Bowel Syndrome · through 2026
Translational Research Center for Gastrointestinal Disorders (TARGID)
Short Bowel Syndrome · through 2026
State Key Laboratory of Primate Biomedical Research
Rett Syndrome · through 2026
State Key Laboratory of Primate Biomedical Research
Rett Syndrome · through 2026
Department of Biochemistry and Microbiology
Rett Syndrome · through 2026
Department of Pharmacology
Rett Syndrome · through 2026
Department of Molecular Pharmacology and Neuroscience
Rett Syndrome · through 2026
Department of Pediatrics & Human Development
Rett Syndrome · through 2026
Faculty of Electronic and Information Engineering
Rett Syndrome · through 2026
Department of Human and Molecular Genetics
Rett Syndrome · through 2026
State Key Laboratory of Cancer Biology
Glioma · through 2026
Department of Neurosurgery
Glioma · through 2026
Department of Neurology
Glioma · through 2026
Department of Neurosurgery
Glioma · through 2026
Department of Clinical Diagnosis
Glioma · through 2026
Otsuka Pharmaceutical Development and Commercialization
High blood pressure · through 2026
Preventative Cardiology
High blood pressure · through 2026
—
High blood pressure · through 2026
Department of Rheumatology and Immunology
Rheumatoid arthritis · through 2026
BGI Research
Rheumatoid arthritis · through 2026
BGI Research
Rheumatoid arthritis · through 2026
Department of Chemistry
Rheumatoid arthritis · through 2026
Laboratory Center
Rheumatoid arthritis · through 2026
Department of Biology
Huntington Disease · through 2026
Department of Neurology
Huntington Disease · through 2026
Chitkara College of Pharmacy
Huntington Disease · through 2026
Sorbonne Université
Huntington Disease · through 2026
Department of Psychiatry
Huntington Disease · through 2025
Centre for Molecular Medicine and Therapeutics
Huntington Disease · through 2025
Department of Child and Adolescent Neurology Mayo Clinic College of Medicine and
Epilepsy · through 2026
Department of Obstetrics
Epilepsy · through 2026
Department of Neurology
Epilepsy · through 2026
Department of Neurology
Epilepsy · through 2026
School of Post-Baccalaureate Medicine
Epilepsy · through 2026
Department of Rheumatology and Immunology
Psoriasis · through 2026
Department of Gastroenterology
Psoriasis · through 2026
Department of Gastroenterology
Psoriasis · through 2026
Department of Dermatology
Psoriasis · through 2026
Department of Dermatology
Psoriasis · through 2026
Department of Dermatology
Psoriasis · through 2026
Institute of Environmental Medicine
Psoriasis · through 2026
Center for Dermatology Research
Psoriasis · through 2026
Key Laboratory of RNA Innovation
Psoriasis · through 2026
Department Traditional Chinese Medicine
Ulcerative colitis · through 2026
Shandong Laboratory of Yantai Drug Discovery
Ulcerative colitis · through 2026
Yunnan Key Laboratory of Dai and Yi Medicines
Ulcerative colitis · through 2026
Jiangsu Key Laboratory For Functional Substance of Chinese Medicine
Ulcerative colitis · through 2026
Department of General Surgery
Ulcerative colitis · through 2026
School of Tropical Agriculture and Forestry
Ulcerative colitis · through 2026
School of Tropical Agriculture and Forestry
Ulcerative colitis · through 2026
College of Food and Pharmacy
Ulcerative colitis · through 2026
Department of Gastroenterology
Ulcerative colitis · through 2026
Department of Gastroenterology
Ulcerative colitis · through 2026
IBD Unit
Ulcerative colitis · through 2026
IBD Unit
Ulcerative colitis · through 2026
Department of Allergy
Asthma · through 2026
Department of Allergy
Asthma · through 2026
Department of Medicine V
Asthma · through 2026
Department of Respiratory and Critical Care Medicine
Asthma · through 2026
Department of Pharmacy
Asthma · through 2026
Department of Neurology
Migraine · through 2026
Department of Neurology
Migraine · through 2026
School of Environment and Chemical Engineering
Migraine · through 2026
Neuroscience Center
Migraine · through 2026
Department of Radiology
Migraine · through 2026
Department of Radiology
Migraine · through 2026
Department of Neurology
Migraine · through 2026
Albert Einstein College of Medicine and Montefiore Headache Center
Migraine · through 2026
Department of Neurology
Migraine · through 2026
Department of Neurology
Myasthenia Gravis · through 2026
Department of Cardiothoracic Surgery
Myasthenia Gravis · through 2026
Department of Medical Sciences
Myasthenia Gravis · through 2026
Analysis Group
Myasthenia Gravis · through 2026
Department of Neurology
Myasthenia Gravis · through 2026
Department of Pharmacy
Myasthenia Gravis · through 2026
Department of Neuroscience
Myasthenia Gravis · through 2026
Department of Neurology
Myasthenia Gravis · through 2026
Department of Pediatric Neurology
Myasthenia Gravis · through 2026
Department of Neurology
Myasthenia Gravis · through 2026
Charité-Universitätsmedizin Berlin
Myasthenia Gravis · through 2026
Department of Clinical Medicine
Myasthenia Gravis · through 2026
Department of Cardiovascular Medicine
Marfan Syndrome · through 2026
Department of Cardiovascular Medicine
Marfan Syndrome · through 2026
Department of Cardiovascular Medicine
Marfan Syndrome · through 2026
Department of Cardiology
Marfan Syndrome · through 2026
Department of Biomedical Engineering
Marfan Syndrome · through 2026
Eye Institute and Department of Ophthalmology
Marfan Syndrome · through 2026
Department of Medical and Molecular Genetics
Ehlers-Danlos Syndrome · through 2026
Wingate Institute of Neurogastroenterology
Ehlers-Danlos Syndrome · through 2026
Department of Surgery
Ehlers-Danlos Syndrome · through 2026
Division of Metabolism
Phenylketonuria · through 2026
Department of Organization and Management in Health Care
Phenylketonuria · through 2026
Department of Pediatrics
Phenylketonuria · through 2026
Istanbul University, Istanbul Faculty of Medicine, Division of Nutrition and Metabolism, I
CTX · through 2026
Department of Pediatric Nutrition and Metabolism, İstanbul University-Cerrahpaşa, Cerrahpa
CTX · through 2026
Department of Women's and Children's Health, Laboratory of Mass Spectrometry and Metabolom
CTX · through 2026
Department of Ophthalmology and Visual Sciences, Kellogg Eye Center, University of Michiga
CTX · through 2026
Division of Neurology, Department of Clinical Medicine, Universidade Federal do Ceará, For
CTX · through 2026
Division of Neurology, Department of Clinical Medicine, Universidade Federal do Ceará, For
CTX · through 2026
Department of Pharmacy and Clinical Pharmacology, Amsterdam UMC Location University of Ams
CTX · through 2026
Hacettepe University, Faculty of Medicine, Department of Pediatrics, Division of Pediatric
CTX · through 2026
CF Therapies
Cystic Fibrosis · through 2026
Nuffield Department of Primary Care Health Sciences
Cystic Fibrosis · through 2026
Department of Pediatrics
Cystic Fibrosis · through 2026
Department of Respiratory and Critical Care Medicine The Affiliated Changsha Cen
Cystic Fibrosis · through 2026
Department of Pediatrics
Cystic Fibrosis · through 2026
Royal Alexandra Children's Hospital
Cystic Fibrosis · through 2026
Leeds Teaching Hospitals NHS Trust
Cystic Fibrosis · through 2026
School of Life Sciences
Cystic Fibrosis · through 2026
Sri Manakula Vinayagar Medical College
Cystic Fibrosis · through 2026
Department of Genetic Medicine
Cystic Fibrosis · through 2026
Hebei Provincial People's Hospital
Thrombotic Thrombocytopenic Purpura · through 2026
Department of Internal Medicine
Thrombotic Thrombocytopenic Purpura · through 2026
Department of Rheumatology and Autoimmunology
Thrombotic Thrombocytopenic Purpura · through 2026
Queensland Brain Institute
Amyotrophic Lateral Sclerosis · through 2026
Department of Nutrition and Dietetics
Amyotrophic Lateral Sclerosis · through 2026
Department of Neurosurgery
Amyotrophic Lateral Sclerosis · through 2026
Department of Physiology
Amyotrophic Lateral Sclerosis · through 2026
Department of Physiology
Amyotrophic Lateral Sclerosis · through 2026
Department of Physiology
Amyotrophic Lateral Sclerosis · through 2026
Department of Neurology
Amyotrophic Lateral Sclerosis · through 2026
Center for ALS and Other Motor Neuron Disorders
Amyotrophic Lateral Sclerosis · through 2026
"Rita Levi Montalcini' Department of Neuroscience
Amyotrophic Lateral Sclerosis · through 2026
"Rita Levi Montalcini' Department of Neuroscience
Amyotrophic Lateral Sclerosis · through 2026
"Rita Levi Montalcini' Department of Neuroscience
Amyotrophic Lateral Sclerosis · through 2026
"Rita Levi Montalcini' Department of Neuroscience
Amyotrophic Lateral Sclerosis · through 2026
Department of Ultrasound
Amyotrophic Lateral Sclerosis · through 2026
Ningxia Key Laboratory of Cerebrocranial Diseases
Multiple sclerosis · through 2026
College of Traditional Chinese Medicine
Multiple sclerosis · through 2026
Translational Imaging in Neurology (ThINK) Basel
Multiple sclerosis · through 2026
Translational Imaging in Neurology (ThINK) Basel
Multiple sclerosis · through 2026
Icahn School of Medicine at Mount Sinai
Multiple sclerosis · through 2026
Department of Medical and Surgical and Advanced Technologies
Multiple sclerosis · through 2026
Department of Neuroradiology
Multiple sclerosis · through 2026
Institute of Environmental Medicine
Multiple sclerosis · through 2026
Neuroimmunology Research Lab
Multiple sclerosis · through 2026
Beijing National Laboratory for Molecular Science (BNLMS)
Gaucher Disease · through 2026
Department of Pediatrics
Gaucher Disease · through 2026
Department of Neurology
Spinal Muscular Atrophy · through 2026
Integrative Program for Biological and Genome Sciences
Spinal Muscular Atrophy · through 2026
Centre for Neuromuscular Diseases
Spinal Muscular Atrophy · through 2026
Centre for Neuromuscular Diseases
Spinal Muscular Atrophy · through 2026
Centre for Neuromuscular Diseases
Spinal Muscular Atrophy · through 2026
Centre for Neuromuscular Diseases
Spinal Muscular Atrophy · through 2026
Department of Neurology & Neurosurgery
Spinal Muscular Atrophy · through 2026
Department of Pharmacy
Pulmonary Tuberculosis · through 2026
Department of Infectious Diseases
Pulmonary Tuberculosis · through 2026
Wenzhou Institute
Pulmonary Tuberculosis · through 2026
Department of Dermatology
Neurofibromatosis, Type 1 · through 2026
First Department of Pediatrics
Neurofibromatosis, Type 1 · through 2026
Department of Surveying and Geoinformatics Engineering
Neurofibromatosis, Type 1 · through 2026
Department of Pediatrics
Neurofibromatosis, Type 1 · through 2026
Department of Clinical Laboratory
Autoimmune Hemolytic Anemia · through 2026
Department of Clinical Laboratory
Autoimmune Hemolytic Anemia · through 2026
Department of Clinical Laboratory
Autoimmune Hemolytic Anemia · through 2026
Division of Pediatric Gastroenterology
Short Bowel Syndrome · through 2026
Division of Pediatric Gastroenterology
Short Bowel Syndrome · through 2026
Department of Digestive Diseases
Short Bowel Syndrome · through 2026
Division of Gastroenterology
Short Bowel Syndrome · through 2026
Division of Gastroenterology
Short Bowel Syndrome · through 2026
Division of Pediatric Surgery
Short Bowel Syndrome · through 2026
University Children's Hospital Tübingen
Short Bowel Syndrome · through 2026
Vanderbilt Kennedy Center
Rett Syndrome · through 2026
Department of Biochemistry and Biotechnology
High blood pressure · through 2026
Division of Nephrology
High blood pressure · through 2026
Department of Research and Evaluation Kaiser Permanente Southern California Pasa
High blood pressure · through 2026
Division of General Medicine Columbia University Irving Medical Center New York
High blood pressure · through 2026
Institute of Sociology
High blood pressure · through 2026
Department of Nephrology
High blood pressure · through 2026
Department of General Practice
High blood pressure · through 2026
National Institute of Nutrition and Health
High blood pressure · through 2026
Migrant Health Research Group
High blood pressure · through 2026
Department of Laboratory Medicine
High blood pressure · through 2026
School of Public Health
High blood pressure · through 2026
Heart Center and Beijing Key Laboratory of Hypertension
High blood pressure · through 2026
Mayo Clinic
High blood pressure · through 2026
The First Affiliated Hospital of Zhejiang Chinese Medical University (Zhejiang P
High blood pressure · through 2026
Faculty of Nursing
High blood pressure · through 2026
Department of Biology
Huntington Disease · through 2026
First Clinical Medical College
Huntington Disease · through 2026
Institute of Molecular Biology
Huntington Disease · through 2026
Department of Obstetrics and Gynecology of the Second Affiliated Hospital
Huntington Disease · through 2026
Centre for Molecular Medicine and Therapeutics
Huntington Disease · through 2026
Department of Neurology
Huntington Disease · through 2026
Department of Biostatistics
Huntington Disease · through 2026
Department of Neurology
Huntington Disease · through 2026
Department of Medical Genetics
Huntington Disease · through 2026
Translational Neuroendocrine Research Unit
Huntington Disease · through 2026
Department of Functional Neurosurgery
Epilepsy · through 2026
Department of Functional Neurosurgery
Epilepsy · through 2026
Department of Child Health Care
Epilepsy · through 2026
Department of Neurology
Epilepsy · through 2026
Department of Child Health Care
Epilepsy · through 2026
Department of Pediatrics
Epilepsy · through 2026
Department of Neurology
Epilepsy · through 2026
Department of Pediatrics
Epilepsy · through 2026
Division of Neurology & Epilepsy
Epilepsy · through 2026
Department of Neurology
Epilepsy · through 2026
Department of Neurology and Neurological Sciences Stanford University Palo Alto
Epilepsy · through 2026
Norcliffe Foundation Center for Integrative Brain Research Seattle Children's Ho
Epilepsy · through 2026
Department of Neurology Boston Children's Hospital Boston Massachusetts USA
Epilepsy · through 2026
Yulin City Center for Disease Control and Prevention (Yulin Health Supervision I
Asthma · through 2026
Department of Trauma Surgery I
Asthma · through 2026
Otolaryngology Department
Asthma · through 2026
Acupuncture and Tuina School
Migraine · through 2026
Department of Cardiology
Migraine · through 2026
Department of Nuclear Medicine
Migraine · through 2026
Clinical Pharmacology
Migraine · through 2026
Helen and Robert Appel Alzheimer's Disease Research Institute
Migraine · through 2026
Brain Health Clinic
Migraine · through 2026
Headache Center
Migraine · through 2026
Neuroscience Center
Migraine · through 2026
ICES
Migraine · through 2026
Department of Pain Management
Migraine · through 2026
School of Traditional Chinese Medicine
Migraine · through 2026
Department of Cardiovascular Medicine
Marfan Syndrome · through 2026
Department of Cardiovascular Medicine
Marfan Syndrome · through 2026
Division of Neurosurgery
Marfan Syndrome · through 2026
Department of Cardiovascular Medicine
Marfan Syndrome · through 2026
Department of Cardiothoracic surgery ICU
Marfan Syndrome · through 2026
Departments of Cardiology and Radiology
Marfan Syndrome · through 2026
Amsterdam Cardiovascular Sciences
Marfan Syndrome · through 2026
Department of Biomolecular Medicine
Marfan Syndrome · through 2026
Department of Cardiovascular Surgery
Marfan Syndrome · through 2026
The Institute of Medical Science
Marfan Syndrome · through 2026
Marfan Syndrome Center
Marfan Syndrome · through 2026
Division of Vascular and Endovascular Surgery
Ehlers-Danlos Syndrome · through 2026
Institute for Genomic Biology and Carle Illinois College of Medicine
Ehlers-Danlos Syndrome · through 2026
Department of Otolaryngology
Ehlers-Danlos Syndrome · through 2026
Carle Illinois College of Medicine
Ehlers-Danlos Syndrome · through 2026
Department of Ophthalmology
Ehlers-Danlos Syndrome · through 2026
Department of General Internal Medicine
Ehlers-Danlos Syndrome · through 2026
Department of Health and Physical Education
Ehlers-Danlos Syndrome · through 2026
Cumming School of Medicine
Ehlers-Danlos Syndrome · through 2026
Lifespan and Population Health
Cystic Fibrosis · through 2026
School of Medicine
Cystic Fibrosis · through 2026
Department of Cardiology
Cystic Fibrosis · through 2026
Department of Microbiology and Immunology
Cystic Fibrosis · through 2026
Liverpool University Hospitals NHS Foundation Trust
Asthma · through 2026
University of Glasgow
Asthma · through 2026
Guy's and St Thomas' Hospitals NHS Trust
Asthma · through 2026
Lineberger Comprehensive Cancer Center
Asthma · through 2026
Department of Neurology University Clinic RWTH Aachen Aachen Germany
Ehlers-Danlos Syndrome · through 2026
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