rare disease

Hong Kong, Shanghai and Florham Park, New Jersey — October 29, 2026 HUTCHMED (China) Limited announced that the US...

Allschwil, Switzerland — October 28, 2026 Idorsia Ltd. announced the initiation of FAB-KLEAR, a pivotal Phase 3 kidney biopsy...

TOKYO, Japan, and CAMBRIDGE, United Kingdom — October 28, 2026 Nxera Pharma Co., Ltd., a technology-powered biopharmaceutical company developing...

HYOGO, Japan — September 28, 2026 JCR Pharmaceuticals Co., Ltd. announced that Japan’s Ministry of Health, Labour and Welfare...

Foster City, California, U.S., September 28, 2026 Mirum Pharmaceuticals has announced a major clinical-development milestone for brelovitug, an investigational...

San Carlos, California, U.S., September 24, 2026 Glycomine, Inc. has announced that the U.S. Food and Drug Administration (FDA)...

LEIDEN, Netherlands — September 25, 2026 Pharming Group N.V. announced that the U.S. Food and Drug Administration (FDA) has...

MALVERN, Pa. — September 25, 2026 Ocugen Inc. announced that its investigational modifier gene therapy OCU400 has received provisional...

New Taipei City, Taiwan, September 22, 2026 Caliway Biopharmaceuticals has announced positive final results from its randomized, single-blind, placebo-controlled...

EMERYVILLE, Calif. — September 24, 2026 Octant, Inc. announced that the first patient has been dosed in the Phase...