GLYCOGEN STORAGE DISEASE TYPE IA
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE IA explained in plain language.
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Gene therapy breakthrough for rare metabolic disease
Disease control Recruiting nowThis study tests a new gene therapy called BEAM-301 for adults with a specific form of glycogen storage disease type Ia (GSDIa), a rare genetic disorder that causes dangerously low blood sugar. The treatment aims to fix a faulty gene to help the body better control blood sugar le…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Phase: PHASE1, PHASE2 • Sponsor: Beam Therapeutics Inc. • Aim: Disease control
Last updated May 12, 2026 13:40 UTC
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10-Year watch: how does DTX401 hold up in GSDIa patients?
Knowledge-focused Recruiting nowThis study follows about 140 people with Glycogen Storage Disease Type Ia who have already received the gene therapy DTX401. Researchers will track safety and how well the treatment works for at least 10 years. No new drug is given; participants simply attend regular check-ups to…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated May 17, 2026 08:01 UTC
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Home test accuracy under the microscope for rare metabolic disease
Knowledge-focused 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: Knowledge-focused
Last updated May 14, 2026 12:04 UTC
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Can a home device replace lab tests for rare disease?
Knowledge-focused Recruiting nowThis study looks at whether portable lactate monitors (finger-stick devices) give accurate results for people with glycogen storage disease type Ia compared to standard lab tests. About 10 participants will have their lactate and glucose levels measured at home and in the lab. Th…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Connecticut Children's Medical Center • Aim: Knowledge-focused
Last updated May 11, 2026 20:39 UTC