Sarepta Therapeutics is meeting with the FDA in June to discuss expanding the use of its Duchenne muscular dystrophy gene ...

American Society of Gene & Cell Therapy meeting sees 10% drop in attendance. Notable absences include FDA officials, David ...

The FDA is prepping to renegotiate its multibillion-dollar, quinquennial user fee agreements with the drug industry later ...

BioMarin plans to buy Boston-based Inozyme Pharma and its Phase 3 enzyme replacement therapy for about $270 million, the ...

The Trump administration is partnering with Mark Cuban’s compounding business and four other entities to improve the ...

Leo Pharma is doing an extensive revamp of its leadership team as it splits product strategy and international operations ...

It's been 13 weeks since Aardvark Therapeutics' $94 million initial public offering on Feb. 13 in the bleakest streak for ...

The government 'did not act contrary to the law' by requiring drugmakers to seek approval before implementing proposed new 340B rebate models, a federal judge ruled.

Rocket Pharmaceuticals reports early success in gene therapy trial for heart condition PKP2-ACM, with 3 patients showing ...