GLYCOGEN STORAGE DISEASE TYPE IA
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE IA explained in plain language.
Never miss a new study
Get alerted when new GLYCOGEN STORAGE DISEASE TYPE IA trials appear
Sign up with your email to follow new studies for GLYCOGEN STORAGE DISEASE TYPE IA, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
Decade-Long watch: tracking gene Therapy's lasting impact on rare metabolic disease
Disease control Recruiting nowThis study will monitor 140 people with Glycogen Storage Disease Type Ia (GSDIa) for at least 10 years after they received an investigational gene therapy called DTX401. The main goal is to understand the long-term safety and effectiveness of the treatment by tracking health outc…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
-
One-Shot gene therapy trial offers hope for rare metabolic disease
Disease control Recruiting nowThis early-stage study is testing a single dose of an experimental gene therapy called BEAM-301 in adults with a specific genetic form of glycogen storage disease type Ia (GSDIa). The main goals are to see if the treatment is safe and to find the best dose. Researchers will measu…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Phase: PHASE1, PHASE2 • Sponsor: Beam Therapeutics Inc. • Aim: Disease control
Last updated Mar 25, 2026 14:08 UTC
-
Can a simple finger prick replace lab tests for rare disease?
Diagnosis Recruiting nowThis study aims to check if home lactate and glucose meters give readings as accurate as hospital lab tests for people with Glycogen Storage Disease (GSD). Ten patients with specific types of GSD will spend about 8 hours in the hospital having blood drawn from their arm and finge…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Connecticut Children's Medical Center • Aim: Diagnosis
Last updated Mar 30, 2026 14:34 UTC
-
Home test kits put to the test for rare metabolic disease
Knowledge-focused Recruiting nowThis study aims to check if portable home lactate and glucose monitors give accurate readings for people with Glycogen Storage Disease Type 1a. Researchers will compare results from finger-prick tests using home devices to standard blood tests drawn in a lab. The goal is to see i…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Connecticut Children's Medical Center • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:41 UTC