About Autosomal dominant adult-onset proximal spinal muscular atrophy
Autosomal dominant adult-onset proximal spinal muscular atrophy is a rare disease catalogued by Orphanet (ORPHA:209335). It is associated with the VAPB 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 Autosomal dominant adult-onset proximal spinal muscular atrophy trials.
Search ClinicalTrials.gov for "Autosomal dominant adult-onset proximal spinal muscular atrophy" or filter by Orphanet code ORPHA:209335 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
Find recruiting Autosomal dominant adult-onset proximal spinal muscular atrophy trials
Search 500,000+ studies from ClinicalTrials.gov, filtered for Autosomal dominant adult-onset proximal spinal muscular atrophy. Updated daily.