rare disease therapy

Boston, Massachusetts, August 11, 2026 Rhythm Pharmaceuticals announced that the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA)...

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

ASHBURN, Va., June 16, 2026 Quoin Pharmaceuticals announced a positive clinical update from its ongoing pediatric Netherton Syndrome (NS)...

PARMA, Italy, June 5, 2026 Chiesi Global Rare Diseases has announced a major regulatory milestone following the European Commission’s...

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...