CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...

Sarepta Therapeutics faces a challenging period as it navigates recent setbacks with its groundbreaking gene therapy, leading ...

The Centers for Medicare and Medicaid Services has named 33 states, as well as Washington, D.C., and Puerto Rico, as participants in its Cell and Gene Therapy Access Model, which is designed to treat ...

This study marks the first time gene therapy has shown consistent success in both children and adults with OTOF-related ...

Sanofi SNY has announced that the FDA has granted a fast-track designation to its one-time intravitreal gene therapy, SAR446597, for the treatment of geographic atrophy (GA) due to age-related macular ...

Paris: Sanofi has received fast track designation from the US Food and Drug Administration (FDA) for SAR446597, a one-time ...

The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...

Sanofi has been granted a speedy review from the FDA for a gene therapy for geographic atrophy (GA), a complication of age-related macular degeneration (AMD), as it prepares to test it in a clinical ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

BETHESDA, Md. (July 16, 2025) – Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million ...

Groundbreaking new research shows that it is possible to use gene therapy to restore hearing in both children and adults.

This is the first time such results have been achieved in both children and adult patients born with a specific type of ...