rare diseases

BOSTON, August 6, 2026 X4 Pharmaceuticals reported second quarter 2026 financial results while highlighting continued progress in the global...

BOSTON, August 4, 2026 Rhythm Pharmaceuticals announced encouraging preliminary Phase 2 clinical trial results for RM-718, its investigational once-weekly...

NEW YORK, N.Y., July 23, 2026 Apertura Gene Therapy has announced its participation in a consortium selected to receive...

ROME & CORAL GABLES, Fla., July 16, 2026 Angelini Pharma has completed its acquisition of Catalyst Pharmaceuticals, marking a...

SAN DIEGO, California, July 13, 2026 Aethlon Medical, Inc. has announced encouraging interim findings from the second patient cohort...

DUBLIN, Ireland, July 13, 2026 Keenova Therapeutics plc has announced the publication of a new peer-reviewed real-world evidence study...

MALVERN, Pa., July 13, 2026 Ocugen announced the signing of a binding term sheet with Roots Pharmaceutical and its...

PRATTELN, Switzerland, July 13, 2026 Santhera Pharmaceuticals announced the expansion of its exclusive distribution agreement with Biomedica, extending commercial...

PHILADELPHIA, Pennsylvania, USA, July 9, 2026 GEMMA Biotherapeutics (GEMMABio) has been awarded funding from the Advanced Research Projects Agency...

Pratteln, Switzerland, July 1, 2026 Santhera Pharmaceuticals announced positive topline findings from a Phase 1 clinical study of AGAMREE®...