rare diseases

question ask wonder who why

Biotech

FDA denies request to reconsider Stealth's ultrarare disease med

The biotech says it's "evaluating all options" after the FDA directed the biotech to resubmit a new drug application for its rare disease candidate.

Gabrielle Masson Aug 8, 2025 1:58pm

pipeline pipes RD research

BioMarin drops PKU drug once seen as possible Palynziq successor

Aug 5, 2025 4:48am

A ballpoint pen rests on top of a stack of documents ready for signing

Senate bill aims to keep NIH, CDC funds intact

Aug 4, 2025 11:30am

Model skeleton in an anatomy classroom

Antibody strengthens bones in mice with brittle bone disease

Jul 30, 2025 2:54pm

FDA stop sign

Fierce Pharma

FDA snubs Ultragenyx gene therapy over manufacturing issues

Jul 14, 2025 10:16am

relay

Rallybio shifts gears, cashing out of Recursion pact

Jul 8, 2025 8:59am