Disease Directory Congenital myopathy with excess of thin filaments
Neuromuscular

Congenital myopathy with excess of thin filaments

Type

Disease

Gene

ACTA1

About Congenital myopathy with excess of thin filaments

Congenital myopathy with excess of thin filaments is a rare disease catalogued by Orphanet (ORPHA:98904). It is associated with the ACTA1 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 Congenital myopathy with excess of thin filaments trials.

Search ClinicalTrials.gov for "Congenital myopathy with excess of thin filaments" or filter by Orphanet code ORPHA:98904 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

Visit website ↗

Orphanet

European reference resource for rare diseases (ORPHA:98904)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting Congenital myopathy with excess of thin filaments trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Congenital myopathy with excess of thin filaments. Updated daily.