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

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

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

D. Boral Capital reaffirmed their buy rating on shares of Anavex Life Sciences (NASDAQ:AVXL – Free Report) in a report published on Wednesday morning,Benzinga reports. D. Boral Capital currently has a ...

Total operating expenses for the fourth quarter of 2024 were $287 million, including non-cash stock-based compensation of $40 ...

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 ...

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

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

Dr. Eric Levine’s cutting-edge autism research at UConn School of Medicine is reprogramming the donated skin or blood cells ...

Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...

Encoded’s layoffs will mostly affect its technology and early-stage research and development functions. The move is expected ...

Ultragenyx's pipeline is rich with promising candidates, with setrusumab for osteogenesis imperfecta (OI) and GTX-102 for Angelman syndrome leading the charge. Setrusumab, currently in Phase 3 ...