The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

The FDA’s accelerated approval pathway plays a vital role, and scrapping it or reversing course on already-approved therapies ...

Ocugen, Inc. (NASDAQ: OCGN) announced its participation in the 2025 BIO International Convention, held from June 16-19 at the ...

Health secretary Robert F. Kennedy Jr. yesterday appointed a new, smaller vaccine advisory committee at the CDC — just two ...

"FDA approves Capsida’s CAP-003 gene therapy for Parkinson’s" was originally created and published by Pharmaceutical ...

Learn more about the generative AI technology that will be used at the Food and Drug Administration (FDA) to speed up reviews ...

The U.S. Food and Drug Administration (FDA) says it plans to use artificial intelligence (AI) to help speed the approval of ...

Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...

This is a developing news story. Please check back soon for updates. The FDA granted VG801 rare pediatric disease designation ...