Ultragenyx Pharmaceutical Inc

This 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…

This 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 …

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…