Disease Directory Permanent neonatal diabetes mellitus-pancreatic and cerebellar agenesis syndrome
Neurological

Permanent neonatal diabetes mellitus-pancreatic and cerebellar agenesis syndrome

Type

Malformation syndrome

Gene

PTF1A

About Permanent neonatal diabetes mellitus-pancreatic and cerebellar agenesis syndrome

Permanent neonatal diabetes mellitus-pancreatic and cerebellar agenesis syndrome is a rare disease catalogued by Orphanet (ORPHA:65288). It is associated with the PTF1A 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 Permanent neonatal diabetes mellitus-pancreatic and cerebellar agenesis syndrome trials.

Search ClinicalTrials.gov for "Permanent neonatal diabetes mellitus-pancreatic and cerebellar agenesis syndrome" or filter by Orphanet code ORPHA:65288 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:65288)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

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Find recruiting Permanent neonatal diabetes mellitus-pancreatic and cerebellar agenesis syndrome trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Permanent neonatal diabetes mellitus-pancreatic and cerebellar agenesis syndrome. Updated daily.