Disease Directory Childhood-onset nemaline myopathy
Neuromuscular

Childhood-onset nemaline myopathy

Type

Disease

Gene

TPM2, MYPN, ACTA1, TPM3, NEB, KBTBD13

About Childhood-onset nemaline myopathy

Childhood-onset nemaline myopathy is a rare disease catalogued by Orphanet (ORPHA:171439). It is associated with the TPM2, MYPN, ACTA1 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 Childhood-onset nemaline myopathy trials.

Search ClinicalTrials.gov for "Childhood-onset nemaline myopathy" or filter by Orphanet code ORPHA:171439 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:171439)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting Childhood-onset nemaline myopathy trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Childhood-onset nemaline myopathy. Updated daily.