Disease Directory Glycogen storage disease due to muscle and heart glycogen synthase deficiency
Metabolic

Glycogen storage disease due to muscle and heart glycogen synthase deficiency

Type

Disease

Gene

GYS1

About Glycogen storage disease due to muscle and heart glycogen synthase deficiency

Glycogen storage disease due to muscle and heart glycogen synthase deficiency is a rare disease catalogued by Orphanet (ORPHA:137625). It is associated with the GYS1 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 muscle and heart glycogen synthase deficiency trials.

Search ClinicalTrials.gov for "Glycogen storage disease due to muscle and heart glycogen synthase deficiency" or filter by Orphanet code ORPHA:137625 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:137625)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting Glycogen storage disease due to muscle and heart glycogen synthase deficiency trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Glycogen storage disease due to muscle and heart glycogen synthase deficiency. Updated daily.