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
-
Gene therapy may free GSDIa patients from constant cornstarch
Disease control CompletedThis phase 3 study tested a gene therapy (DTX401) in 49 people with glycogen storage disease type Ia, a rare condition that requires frequent cornstarch doses to prevent dangerously low blood sugar. The goal was to see if the treatment could reduce or eliminate the need for corns…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 17, 2026 08:30 UTC
-
Gene therapy for rare Sugar-Storage disease shows promise in Long-Term safety check
Disease control CompletedThis study checked the long-term safety of a one-time gene therapy (DTX401) in 12 adults with glycogen storage disease type Ia, a rare condition that causes dangerously low blood sugar. Participants were followed for side effects and how well their bodies handled fasting without …
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE IA
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 17, 2026 08:19 UTC