The upcoming Clinical Trials in Rare Diseases conference will cover the most concerning challenges in rare disease research.

The deal is part of a wider $55bn investment strategy by J&J to bolster US manufacturing amid pressure from Trump.

Before Novo arrived as its owner, issues at the Bloomington facility caused a two-month delay for Eylea HD’s FDA approval decision in 2023. In November 2024, the FDA also found quality control lapses ...

Keros received orphan drug designation from the US FDA for its investigational therapy KER-065 to treat Duchenne muscular dystrophy (DMD).

The National Institute for Health and Care Excellence (NICE) has recommended a combination of Padcev and Keytruda for the ...