Sarepta Therapeutics’ long-awaited confirmatory study of two of its ‘exon-skipping’ therapies for forms of Duchenne muscular dystrophy failed to meet its primary endpoint, the company

Two federal district courts have reached opposite conclusions in cases challenging states’ authority to regulate how drugmakers distribute 340B discounts. An Oklahoma federal judge on

uniQure has revealed that the FDA has said it is not prepared to review its Huntington’s gene therapy in what seems to be a change

Eli Lilly plans to build another facility to make its oral GLP-1 drug orforglipron. The $3 billion site will be located in Katwijk, the Netherlands.

The UK’s medicines regulatory agency said it is working to “overhaul” its framework for approving rare disease therapies. The Medicines and Healthcare products Regulatory Agency