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 ...

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

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

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

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

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 ...

California Institute for Regenerative Medicine (CIRM) has awarded a $5.8 million translational research grant to a scientist at the University of California, Los Angeles (UCLA) to further support the ...