The FDA proposed new guidelines for approving experimental therapies for rare diseases without large group studies.

The advent of AI at the US Food and Drug Administration (FDA) is changing the complexion of the regulatory approval process for medical devices. In May, FDA commissioner Dr Martin Makary announced ...

The drug was finally approved in September 2025 through the FDA’s accelerated approval pathway, based on improvement in knee extensor muscle strength, for adults and children with Barth syndrome who ...