Disease Directory Hypomyelination with atrophy of basal ganglia and cerebellum
Rare Disease

Hypomyelination with atrophy of basal ganglia and cerebellum

Type

Disease

Gene

TUBB4A, UFM1

About Hypomyelination with atrophy of basal ganglia and cerebellum

Hypomyelination with atrophy of basal ganglia and cerebellum is a rare disease catalogued by Orphanet (ORPHA:139441). It is associated with the TUBB4A, UFM1 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 Hypomyelination with atrophy of basal ganglia and cerebellum trials.

Search ClinicalTrials.gov for "Hypomyelination with atrophy of basal ganglia and cerebellum" or filter by Orphanet code ORPHA:139441 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:139441)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting Hypomyelination with atrophy of basal ganglia and cerebellum trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Hypomyelination with atrophy of basal ganglia and cerebellum. Updated daily.