rare diseases

Combine

More collaborative FDA can accelerate rare disease R&D: report

The National Academies is recommending the FDA and European Medicines Agency implement a “navigation service” to advise on regulatory pathways.

Nick Paul Taylor Sep 13, 2024 5:00am

curtains spotlight show performance

AAV gene therapy biotech unveils with 10 pipeline products

Sep 12, 2024 7:00am

Space rocket moon explore

Novartis ignites new stage of Voyager pact with $15M capsid deal

Sep 5, 2024 9:45am

MA strategy deal block push come together form

Cyclo Therapeutics merges with longtime backer Rafael Holdings

Aug 22, 2024 8:50am

Photo of a carnival ride that swings people in elevated chairs caught in motion

BridgeBio’s offshoot GondolaBio launches with $300M

Aug 21, 2024 11:36am

singer Celine Dion performing on the Eiffel Tower during the opening ceremony of the Paris 2024 Olympic Games

In the wake of Céline Dion's comeback, SPS marches toward a cure

Aug 15, 2024 2:45pm