Sarepta Therapeutics and partner Roche have reported a second case of fatal liver failure with their Duchenne muscular dystrophy (DMD) gene therapy Elevidys and stopped using it to treat some ...

Faruqi & Faruqi, LLP Securities Litigation Partner James (Josh) Wilson Encourages Investors Who Suffered Losses Exceeding ...

The FDA has launched a formal investigation into two liver failure-related deaths in patients treated with Duchenne gene therapy Elevidys.

A journey through the FDA’s newly released complete response letters gave glimpses into the journeys to market for Eli ...

The deal gives AstraZeneca’s rare disease unit Alexion access to specialized capsids developed by the Japanese biotech JCR ...

The U.S. Food and Drug Administration has declined to approve Capricor Therapeutics' cell therapy for a heart condition and ...

Capricor Therapeutics (CAPR) on Friday said the U.S. Food and Drug Administration (FDA) has refused to approve its lead cell ...

Discover a study highlighting the high prevalence of MASLD and liver fibrosis in patients with type 2 diabetes using ...

It's a disease that impacts nearly a quarter of all adults across the U.S., and you may not even know you have it since many ...

Discover a study revealing how genetic and advanced diagnostics clarify the causes and outcomes of paediatric acute liver failure in Germany.

Finerenone gained FDA approval for heart failure treatment, showcasing significant benefits in reducing cardiovascular events ...

FDA researchers report that the upper end of reported consumer use (~400mg of daily CBD intake) of cannabidiol (CBD) may ...