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

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

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 ...

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

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 ...

- ZEVASKYN is the only FDA-approved therapy to treat RDEB wounds with a single application - ...

Nanoscope Therapeutics submitted the first modules of a biologics license application for MCO-010, an investigational gene ...

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

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