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

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...

Sarepta Therapeutics said on Wednesday it would lay off 36% of its workforce, or about 500 employees, after the recent deaths of two patients who had received its gene therapy Elevidys.

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

The company is also pausing research for several treatments it has been developing for another form of muscular dystrophy.

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 ...

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

Sarepta Therapeutics said on Wednesday it would lay off 36% of its workforce, or about 500 employees, after two recent deaths of patients who had received its gene therapy, Elevidys. Shares of the ...

The model was initially launched by the Biden administration in February 2023, and CMS announced that SCD would be the first focus of the model the following January. This is the ...

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

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 ...