States representing about 84% of Medicaid beneficiaries are participating in an initiative that enables the U.S. government ...

This study marks the first time gene therapy has shown consistent success in both children and adults with OTOF-related deafness. It also paves the way for similar therapies targeting other genes, ...

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 ...

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

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

A single shot of gene therapy restored hearing in people born deaf, with most showing improvement within weeks and some ...

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

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral ...

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

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer ...