GLYCOGEN STORAGE DISEASE TYPE IA
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE IA explained in plain language.
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10-Year watch begins for GSDIa gene therapy patients
Disease control Recruiting nowThis study follows people with Glycogen Storage Disease Type Ia (GSDIa) who have already received the gene therapy DTX401. Researchers will monitor their health and how well the treatment works for at least 10 years. The goal is to track side effects, pregnancy outcomes, and chan…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 10:51 UTC
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Gene therapy breakthrough for rare Sugar-Storage disease
Disease control Recruiting nowThis study tests a new gene therapy called BEAM-301 in 36 adults with a specific form of glycogen storage disease type Ia. The goal is to see if it is safe and can help control blood sugar levels, reducing the need for frequent cornstarch doses. Participants must have a confirmed…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Phase: PHASE1, PHASE2 • Sponsor: Beam Therapeutics Inc. • Aim: Disease control
Last updated May 26, 2026 10:40 UTC
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Home meters put to the test for rare disease monitoring
Diagnosis Recruiting nowThis study aims to see if a home lactate meter and a standard glucose meter give accurate readings compared to lab tests in people with glycogen storage disease types Ia, Ib, and XI. Ten participants will spend about 8 hours in the hospital for hourly blood draws and finger-stick…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Connecticut Children's Medical Center • Aim: Diagnosis
Last updated May 22, 2026 13:52 UTC
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Can a simple finger-stick replace lab tests for rare metabolic disease?
Knowledge-focused Recruiting nowThis study looks at whether portable lactate monitors (like those used at home) give accurate results for people with glycogen storage disease type Ia compared to standard lab tests. Ten participants will have their lactate and glucose levels checked using both a finger-stick dev…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Connecticut Children's Medical Center • Aim: Knowledge-focused
Last updated May 26, 2026 10:48 UTC