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🔗 CustomizeMedical Overview of Congenital Muscular Dystrophy Due To Integrin Alpha-7 Deficiency
Sources citedCongenital muscular dystrophy with integrin alpha-7 deficiency is a rare, genetic, congenital muscular dystrophy due to extracellular matrix protein anomaly characterized by early motor development delay and muscle weakness with mild elevation of serum creatine kinase, that may be followed by progressive disease course with predominantly proximal muscle weakness and atrophy, motor development regress, scoliosis and respiratory insufficiency.
Classification & codes: GARD 0012587 · Orphanet ORPHA:34520 · OMIM 613204 · ICD-10 G71.2
Sources: GARD (NIH/NCATS), Orphanet · last reviewed 2026. Educational only — not medical advice.
News & Updates
ALYFTREK shows positive Phase 3 results in children ages 2–5
Vertex plans global regulatory submissions for this pediatric age group in the first half of 2026.
Airway clearance routines that actually work for teens
3 open trials match this profile
Locations in NC, FL and GA.
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Care & management overview — Congenital Muscular Dystrophy Due To Integrin Alpha-7 Deficiency
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Congenital Muscular Dystrophy Due To Integrin Alpha-7 Deficiency News & Developments
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Companies Developing Treatments
Biopharma companies with registered trials for Congenital Muscular Dystrophy Due To Integrin Alpha-7 Deficiency — from ClinicalTrials.gov. Informational, not an endorsement, and not every program is in trials.
Preferences only — saved to your account, never shared or sold. No PHI. Sources: GARD (NIH/NCATS), Orphanet.
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Mental Health Toolkit
Sources citedCoping strategies, how to find a a specialist therapist, and mental-health resources built for the ups and downs of living with Congenital Muscular Dystrophy Due To Integrin Alpha-7 Deficiency — for patients and caregivers alike.
Preferences only — saved to your account, never shared or sold. No PHI. Sources: GARD (NIH/NCATS), Orphanet.
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Certified Centers of Excellence
CFF networkCare centers and specialists for Congenital Muscular Dystrophy Due To Integrin Alpha-7 Deficiency, from Tomeko’s verified provider directory (CMS NPPES).
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Sources: GARD (NIH/NCATS), Orphanet · last reviewed 2026. Educational only — not medical advice.