Disease Directory Glycogen storage disease due to glycogen branching enzyme deficiency, childhood neuromuscular form
Metabolic

Glycogen storage disease due to glycogen branching enzyme deficiency, childhood neuromuscular form

Type

Clinical subtype

Gene

GBE1

About Glycogen storage disease due to glycogen branching enzyme deficiency, childhood neuromuscular form

Glycogen storage disease due to glycogen branching enzyme deficiency, childhood neuromuscular form is a rare disease catalogued by Orphanet (ORPHA:308698). It is associated with the GBE1 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 Glycogen storage disease due to glycogen branching enzyme deficiency, childhood neuromuscular form trials.

Search ClinicalTrials.gov for "Glycogen storage disease due to glycogen branching enzyme deficiency, childhood neuromuscular form" or filter by Orphanet code ORPHA:308698 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:308698)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

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Find recruiting Glycogen storage disease due to glycogen branching enzyme deficiency, childhood neuromuscular form trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Glycogen storage disease due to glycogen branching enzyme deficiency, childhood neuromuscular form. Updated daily.