Ultragenyx Pharmaceutical Inc

This study followed 12 adults with glycogen storage disease type Ia (GSDIa) who had received a single dose of gene therapy (DTX401) in an earlier trial. The main goal was to see if the treatment was safe over the long term. Researchers also checked how well it helped control bloo…

This study tested a one-time gene therapy (DTX401) in 49 people with glycogen storage disease type Ia, a rare condition that requires frequent cornstarch to prevent dangerously low blood sugar. The goal was to see if the treatment could reduce or eliminate the need for cornstarch…

This 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 multiple injections into the spine. Researchers monit…