The first few months of 2025 brought hope to the rare disease community. Food and Drug Administration leadership promised ...

The FDA recommends that companies use overall survival as a primary endpoint for clinical trials where feasible. The new ...

The FDA said it has begun publishing reports of adverse events concerning drugs on a daily basis, instead of quarterly, as it ...

The FDA has agreed to speed up a review of a rare disease drug developed by Stealth BioTherapeutics, a notable step after the ...

The U.S. Food and Drug Administration has denied approval for PTC Therapeutics' oral medication for a rare genetic disorder ...