The Recommended Uniform Screening Panel now includes DMD and MLD, which has implications for firms developing treatments for ...

Sarepta Therapeutics recently refinanced a significant portion of its debt, extending maturities and enhancing financial ...

Doug Ingram of Sarepta Therapeutics is my pick for the Worst Biopharma CEO of 2025. He was an easy selection, but unlike some ...

Investor attention has returned to Sarepta Therapeutics amid reports of the biotech firm's vigorous legal strategy to protect ...

Solid Biosciences Inc. (NASDAQ:SLDB) develops therapies for neuromuscular and cardiac diseases in the US. In addition, the ...

Scotland’s NHS can now offer givinostat to ambulant children with Duchenne muscular dystrophy, marking a step forward in care ...

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would cover the $2 million price tag.

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Wedbush upgrades Sarepta Therapeutics, citing overdone selloff and potential upside as FDA could re-approval Elevidys for non ...

This article has been revised and clarified. Pasadena-based biopharma company Arrowhead Pharmaceuticals Inc. had a ...