Concludes Strategic Alternatives Review and Provides Update on Development of KER-065LEXINGTON, Mass., June 09, 2025 (GLOBE ...

Sarepta Therapeutics (NasdaqGS:SRPT) saw its share price rise by 19% over the past month, a move that notably outpaced the broader market's 1% increase for the week and the 13% gain over the year.

JAR of Hope helps support a team of researchers and doctors who are aiming to eliminate Duchenne muscular dystrophy – a fatal ...

Explore REGENXBIO's RGX-121 gene therapy for Hunter syndrome, with an FDA decision due Nov 9, 2025. Click here to read my ...

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

Sarepta is the first company to publicly disclose that it's received the designation, for which the FDA had issued a draft guidance last year.

Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

The campaign to commemorate the work of the Muscular Dystrophy Association was created in partnership with agency Yes&.

Senator Lisa Blunt Rochester (D-Del), Senator Thom Tillis (R-NC) and the Foundation to Eradicate Duchenne recognized as ...

The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug ...