About Severe congenital nemaline myopathy
Severe congenital nemaline myopathy is a rare disease catalogued by Orphanet (ORPHA:171430). It is associated with the ACTA1, NEB, KLHL40 genes. 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 Severe congenital nemaline myopathy trials.
Search ClinicalTrials.gov for "Severe congenital nemaline myopathy" or filter by Orphanet code ORPHA:171430 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 Severe congenital nemaline myopathy trials
Search 500,000+ studies from ClinicalTrials.gov, filtered for Severe congenital nemaline myopathy. Updated daily.