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

One day after stepping down as CDER Director, George Tidmarsh has said he will fight the investigations into his conduct and is reconsidering his decision.

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

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