Ultragenyx Pharmaceutical Inc

This 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…

This study tested the safety of an experimental drug called GTX-102 in children with Angelman syndrome, a rare genetic disorder that affects development. Researchers gave the drug through spinal injections to 74 children to see how well they tolerated it and to measure drug level…

This 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…