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New York, Jan. 14, 2026 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) is proud to announce the recipients of the 2026 MDA Legacy Awards, recognizing excellence in scientific and...

Rare Pediatric Disease Priority Review Voucher (PRV) granted by FDA at approval to be transferred from Sentynl Therapeutics to Cyprium Cyprium eligible to receive tiered royalties and up to $129...

Approval is supported by data from the DEVOTE study which showed the benefit of the SPINRAZA 50 mg and 28 mg regimen in both treatment-naïve and previously-treated nusinersen patients with SMA1Biogen...

Agios today announced its 2026 strategic priorities and key milestones anticipated during the year.

CORAL GABLES, Fla., Jan. 08, 2026 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing,...

Tisento's global Phase 2b PRIZM study, which is evaluating zagociguat for the treatment of the rare mitochondrial disease MELAS, is fully enrolled

Dublin, Jan. 06, 2026 (GLOBE NEWSWIRE) -- The "Curative Therapeutics Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, 2020-2030F" has been added to ResearchAndMarkets.com's...

CORAL GABLES, Fla., Jan. 05, 2026 (GLOBE NEWSWIRE) -- The University of Notre Dame and Catalyst Pharmaceuticals, Inc. (“Catalyst” or “Company”) (Nasdaq: CPRX), a commercial-stage biopharmaceutical...

Agios today announced that the U.S. FDA has approved AQVESME™ (mitapivat), an oral pyruvate kinase (PK) activator, for thalassemia

The FSR Voice of the Patient report outlines a clear path forward for drug developers, researchers, clinicians, and regulators for sarcoidosis.