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Scientists launch Largest-Ever study of Ultra-Rare GSDs

NCT ID NCT06795152

Recruiting now Knowledge-focused Sponsor: Duke University Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tracks 200 people with ultra-rare glycogen storage diseases (GSDs) like types 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome, and Danon disease. Researchers will collect medical records and other data to see how these conditions progress over time. No treatments or drugs are being tested—the goal is simply to learn more about these rare diseases.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

Danon disease disorder of glycogen metabolism glycogen storage disease due to aldolase A deficiency glycogen storage disease due to muscle and heart glycogen synthase deficiency glycogen storage disease due to muscle beta-enolase deficiency glycogen storage disease due to phosphoglycerate mutase deficiency glycogen storage disease VII glycogen storage disorder due to hepatic glycogen synthase deficiency polyglucosan body myopathy 1 with or without immunodeficiency polyglucosan body myopathy type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DANON DISEASE GLYCOGEN STORAGE DISEASE GSD TYPE 0A GSD TYPE 0B GSD VII GSD X GSD XII GSD XIII GSD XV PGBM2 POLYGLUCOSAN BODY MYOPATHY TYPE 1 POLYGLUCOSAN BODY MYOPATHY TYPE 2 PRKAG2 RBCK1 DEFICIENCY TARUI DISEASE

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Duke University

    RECRUITING

    Durham, North Carolina, 27710, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact