rare disease therapy

WASHINGTON, May 27, 2026 Vanda Pharmaceuticals Inc. has announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has...

Cambridge, Massachusetts, USA, May 22, 2026 Agios Pharmaceuticals has announced that the European Commission (EC) has granted marketing authorization...

Leiden, Netherlands, May 22, 2026 Pharming Group N.V. has announced that the European Commission (EC) has granted Marketing Authorization...

PRAGUE, Czech Republic, May 12, 2026 AstraZeneca and Alexion announced positive Phase III clinical trial results for eneboparatide (AZP-3601),...

DURHAM, N.C., May 11, 2026 AskBio Inc., a gene therapy company wholly owned and independently operated as a subsidiary...

BOULDER, Colo., May 7, 2026 Edgewise Therapeutics reported first quarter 2026 financial results while highlighting major progress across its...

Leiden, the Netherlands, May 7, 2026 Pharming Group N.V. announced a major scientific presence at the 2026 Annual Meeting...

PALO ALTO, CA — March 11, 2026 BridgeBio Pharma announced that its investigational therapy BBP-418 demonstrated consistent efficacy and...

Ashburn, Virginia — March 9, 2026 Clinical-stage specialty biopharmaceutical company Quoin Pharmaceuticals Ltd. continues to advance the development of...