As one FDA initiative works to sustain the momentum of rare disease research, other events threaten to slow progress.

The prefilled syringe formulation is approved in the US for the vaccination of adults aged 50 and older,and individuals aged 18 and above at greater risk. Credit: Spino Kim/Shutterstock.com. The US ...

Sarepta Therapeutics is set to cut 500 jobs as part of its restructuring and pipeline prioritisation plan, which is expected to result in annual cash cost savings of $120m in 2026. With its pipeline ...

Due to concerns surrounding its effectiveness, the US Food and Drug Administration (FDA) ended the Rare Pediatric Disease Designation (RPDD) programme in late 2024.