Ultragenyx Pharmaceutical Inc
Clinical trials sponsored by Ultragenyx Pharmaceutical Inc, explained in plain language.
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New spinal injection therapy tested in children with rare genetic disorder
Disease control CompletedThis early-stage clinical trial tested the safety and tolerability of an experimental drug called GTX-102 in children with Angelman syndrome. The drug was given as multiple injections into the spinal fluid. The main goal was to see how well children handled the treatment and to m…
Phase: PHASE1, PHASE2 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Apr 03, 2026 14:42 UTC
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One-Shot gene therapy aims to free patients from constant cornstarch dependence
Disease control CompletedThis study tested a one-time gene therapy injection called DTX401 for people with Glycogen Storage Disease Type Ia (GSDIa), a rare genetic disorder. The main goal was to see if the therapy could safely reduce or eliminate the need for patients to constantly eat cornstarch to keep…
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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Gene therapy patients monitored for years in rare disease study
Disease control CompletedThis study followed 12 adults with a rare genetic liver disorder (GSDIa) for 4 to 6 years after they received a single dose of an experimental gene therapy. The main goal was to check for any long-term safety issues from the treatment. Researchers also monitored whether the thera…
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC