Ultragenyx Pharmaceutical Inc
Clinical trials sponsored by Ultragenyx Pharmaceutical Inc, explained in plain language.
Biopharmaceutical company developing treatments for rare and ultra-rare diseases.
Trade on eToro (ad)Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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Gene therapy could free GSD ia patients from constant cornstarch
Disease control CompletedThis Phase 3 trial tested a gene therapy called DTX401 in 49 people with glycogen storage disease type Ia (GSD Ia). The goal was to see if a single infusion could reduce or eliminate the need for frequent cornstarch doses to keep blood sugar stable. Participants were randomly ass…
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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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. Researchers monitored side effects and how well the therapy helped control blood sugar levels …
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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New angelman drug shows promise in early safety trial
Disease control CompletedThis study tested a new medicine called GTX-102 in 74 children with Angelman Syndrome, a rare genetic disorder causing developmental delays and seizures. The main goal was to see if the drug is safe and tolerable when given as a spinal injection. Researchers monitored side effect…
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC