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

Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

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 ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College ...

Determining which patients should receive the treatment will still require individual risk-benefit assessments, experts say.

With notable therapies from Biogen, Sarepta and MacroGenics failing to show efficacy in pivotal or confirmatory trials, experts question the use of biomarker evidence for approval while one former ...

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 ...