rare disease

BOSTON, August 24, 2026 Rhythm Pharmaceuticals, Inc. announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted...

Warren, New Jersey, August 24, 2026 PTC Therapeutics, Inc. announced that new clinical and real-world evidence for Sephience™ (sepiapterin)...

Spring House, Pennsylvania, U.S. | August 24, 2026 Johnson & Johnson announced that the U.S. Food and Drug Administration...

Waltham, Massachusetts, USA, and San Diego, California, USA, August 21, 2026 Werewolf Therapeutics and Ambros Therapeutics have announced a...

Philadelphia, August 20, 2026 iECURE announced that updated clinical data from the ongoing OTC-HOPE trial of ECUR-506 in infants...

Novato, California, August 19, 2026 Ultragenyx Pharmaceutical Inc. has announced that the U.S. Food and Drug Administration (FDA) granted...

CARDIFF, U.K., August 19, 2026 Biodexa Pharmaceuticals PLC (Nasdaq: BDRX) announced a major recruitment milestone for its Serenta registrational...

TARRYTOWN, N.Y., August 19, 2026 Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved...

PASADENA, Calif., August 17, 2026 Arrowhead Pharmaceuticals, Inc. announced that people living with familial chylomicronaemia syndrome (FCS) in Germany...

Copenhagen, Denmark — August 17, 2026 Evaxion A/S has expanded and refocused its cancer vaccine pipeline with EVX-05, an...