Disease Directory Familial dysautonomia
Rare Disease

Familial dysautonomia

Type

Disease

Gene

ELP1

About Familial dysautonomia

Familial dysautonomia is a rare disease catalogued by Orphanet (ORPHA:1764). It is associated with the ELP1 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 Familial dysautonomia trials.

Search ClinicalTrials.gov for "Familial dysautonomia" or filter by Orphanet code ORPHA:1764 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:1764)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting Familial dysautonomia trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Familial dysautonomia. Updated daily.