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

Reports Q4 revenue $$227.00M, consensus $140.95M.”With the recent launch of our first independent medicine, TRYNGOLZA for familial ...

International Angelman Day is recognized on February 15. The date reflects the genetic defect Angelman Syndrome causes in the 15th chromosome and nods to Feburary’s designation as Rare Disease Month.

For the last several months, and throughout the majority of the NFL season, the Kansas City skyline has illuminated a deep cardinal red and golden hue in honor of the Chiefs. However, things will ...

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

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 ...

Encoded Therapeutics is shrinking its headcount by 29% as the gene therapy biotech draws resources away from its early-stage ...

Rare diseases have become an attractive target for pharmaceutical companies both in Australia and internationally with ...

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.

Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...

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