HUTCHMED Wins FDA Orphan Status for Sovleplenib
Hong Kong, Shanghai, China, and Florham Park, New Jersey, USA, November 5, 2026 HUTCHMED (China) Limited has announced that...
rare diseases
Advertisement
BioMarin Licenses Next-Gen Fabry Disease Therapy Worldwide
SAN RAFAEL, California, October 29, 2026 BioMarin Pharmaceutical Inc. has entered into an exclusive worldwide licensing agreement with GC...
US WorldMeds Acquires Orbus to Advance Glioma Drug Research
LOUISVILLE, Kentucky, and MENLO PARK, California, October 27, 2026 US WorldMeds Ventures, LLC has acquired Orbus Therapeutics, Inc., through...
Syndax’s Revuforj Wins 2026 Prix Galien Rare Disease Award
New York, New York — October 30, 2026 Syndax Pharmaceuticals, Inc. announced that Revuforj® (revumenib) has received the 2026...
Agios Sets February 2027 FDA Goal Date for Mitapivat in Sickle Cell Disease
Cambridge, Massachusetts — October 30, 2026 Agios Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has...
Advertisements
Arrowhead Secures UK Access for REDEMPLO in FCS
Pasadena, California, United States, October 27, 2026 Arrowhead Pharmaceuticals has announced a significant regulatory and healthcare access milestone for...
Tangram Therapeutics Doses First Participant in Phase 1 TGM-148 Clinical Trial
London, UK — October 29, 2026 Tangram Therapeutics announced that the first participant has been dosed in its Phase...
Novellia Study Reveals Gaps in Ehlers-Danlos Patient Records
New York, New York, U.S., October 26, 2026 Novellia has reported new research showing that healthcare systems typically miss...
Apertura and Lexeo Advance Gene Therapy for Friedreich Ataxia
New York, New York, September 23, 2026 Apertura Gene Therapy announced that it will provide Lexeo Therapeutics with access...
Kazia Advances Paxalisib in Rare Seizure Disorders
Sydney, Australia, September 21, 2026 Kazia Therapeutics Limited has announced that its development partner Sovargen Co., Ltd. has dosed...
Advertisement
