Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

Initiation of Phase 1b study using SGT-212 for the treatment of patients with Friedreich's Ataxia expected in 2H25. Click ...

Company Anticipates Submitting for U.S. Accelerated Approval in H1 2026 -WALTHAM, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, ...

Catalyst Pharmaceuticals, Inc. (NASDAQ: CPRX) recently disclosed in an 8-K filing with the Securities and Exchange Commission on January 13, 2025, that the company has posted a corporate presentation ...

Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...

The company says it has in total “nearly 3,000 people at 11 locations in Maine, Connecticut, California, Japan and China, and ...

Phoenix Pinnacle will never forget the impact the kid in the wheelchair had during its greatest stretch in Arizona high ...

We recently published a list of the 12 Biotech Stocks with the Biggest Upside Potential. In this article, we are going to ...

The FDA established its accelerated approval program in 1992 to allow drugs to reach the market quickly for life-threatening ...

Of the 24 drugs, three approvals deviated from the others -- aducanumab; the synthetic hormone hydroxyprogesterone caproate ...

Santhera has begun launch preparations for AGAMREE® in UK with first sales expected in this quarterPratteln, Switzerland, January 16, 2025 – ...