Teen whose story touched Post readers gets up-close look at planes and hangars, joining his friends for ‘special’ visit he ...

Jefferies analysts said these detailed safety outcomes confirm the gene therapy's positive risk/benefit profile in ambulatory ...

DYNE-251 received FDA breakthrough therapy designation, showcasing its potential for patients with Duchenne muscular ...

COALITION Duchenne's 13th annual Expedition Mt Kinabalu will start tomorrow from Shangri-La's Tanjung Aru Resort and Spa, ...

The Health Resources and Services Administration is requesting public comments on its recommendation to add metachromatic ...

Shares of several biotech firms fell in premarket trading Monday after reports that ousted U.S. Food and Drug Administration vaccine regulator Vinay Prasad will return to the agency.

After two patients who received the investigational CDC7 blocker died, pushing forward with SGR-2921’s development would be ...

Pratteln, Switzerland, 13 August, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive agreement with Gen Ilaç ve Saglik Ürünleri San. ve Tic. A.S. (GEN) for the ...

A well-connected drug company and Laura Loomer wanted Kennedy ally Vinay Prasad gone. Trump Chief of Staff Susie Wiles got ...

University of Manchester scientists have mapped the mutations in the tiny protein chains that cause a subtype of muscular ...

SAN DIEGO, Aug. 11, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today ...

Sarepta Therapeutics is selling off Arrowhead Pharmaceuticals’ shares to stretch its cash runway, while also inking a share ...