Feb. 15 is International Angelman Syndrome Day, and the Synk family aims to raise awareness and honor people with the rare ...

Children and adults with Angelman syndrome typically experience balance issues and motor impairment and may have debilitating seizures. They may not be able to walk or speak, and disruptive sleep also ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

Angelman Syndrome is a very rare neurogenetic disorder that occurs in approximately one in 15,000 births, caused by the loss ...

Every morning, eight-year-old Isabelle watches from the window as her two sisters Madison and Olivia leave to walk to school. “Izzy gets so excited, because she thinks she’s going to go. But then, she ...

Emil Kakkis; President, Chief Executive Officer, Director; Ultragenyx Pharmaceutical Inc Erik Harris; Executive Vice President, Chief Commercial Officer; Ultragenyx Pharmaceutical Inc Howard Horn; ...

Good afternoon, and welcome to the Ultragenyx Fourth Quarter and Full Year 2024 Financial Results Conference Call. At this time, all participants are in a listen-only-mode. At the end of the prepared ...