rare diseases

pipeline pipes RD research
Biotech

BioMarin drops PKU drug once seen as possible Palynziq successor

BioMarin is continuing to slim down its pipeline, this time ending work on a preclinical PKU drug once touted as a potential successor to Palynziq.
James Waldron 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
Favicon 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
Graphic image of an arrow nailing the bullseye of a target

Cogent hits primary endpoint, building case for Blueprint rival

Jul 7, 2025 9:16am