Disease Directory DYNC1H1-related autosomal dominant childhood-onset proximal spinal muscular atrophy
Neuromuscular

DYNC1H1-related autosomal dominant childhood-onset proximal spinal muscular atrophy

Type

Etiological subtype

Gene

DYNC1H1

About DYNC1H1-related autosomal dominant childhood-onset proximal spinal muscular atrophy

DYNC1H1-related autosomal dominant childhood-onset proximal spinal muscular atrophy is a rare disease catalogued by Orphanet (ORPHA:209341). It is associated with the DYNC1H1 gene. Search below for active recruiting clinical trials from ClinicalTrials.gov, or visit the Orphanet expert page for specialist centres, natural history studies, and patient registries.

Clinical Trial Eligibility Tips

What to know before applying to DYNC1H1-related autosomal dominant childhood-onset proximal spinal muscular atrophy trials.

Search ClinicalTrials.gov for "DYNC1H1-related autosomal dominant childhood-onset proximal spinal muscular atrophy" or filter by Orphanet code ORPHA:209341 to find disease-specific studies.

Ask your specialist to refer you to a Centre of Expertise — Orphanet lists all accredited centres for this condition worldwide.

Natural history studies and patient registries often enrol before drug trials open — contact the Orphanet patient community first.

Patient Resources

Patient Organization

Orphanet

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Orphanet

European reference resource for rare diseases (ORPHA:209341)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

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Find recruiting DYNC1H1-related autosomal dominant childhood-onset proximal spinal muscular atrophy trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for DYNC1H1-related autosomal dominant childhood-onset proximal spinal muscular atrophy. Updated daily.