Disease Directory Severe intellectual disability and progressive spastic paraplegia
Rare Disease

Severe intellectual disability and progressive spastic paraplegia

Type

Disease

Gene

AP4M1, AP4E1, AP4B1, AP4S1

About Severe intellectual disability and progressive spastic paraplegia

Severe intellectual disability and progressive spastic paraplegia is a rare disease catalogued by Orphanet (ORPHA:280763). It is associated with the AP4M1, AP4E1, AP4B1 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 intellectual disability and progressive spastic paraplegia trials.

Search ClinicalTrials.gov for "Severe intellectual disability and progressive spastic paraplegia" or filter by Orphanet code ORPHA:280763 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

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Orphanet

European reference resource for rare diseases (ORPHA:280763)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

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Find recruiting Severe intellectual disability and progressive spastic paraplegia trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Severe intellectual disability and progressive spastic paraplegia. Updated daily.