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After phase 3 clean sweep, Roche plans oral BTK filing in MS
Roche is preparing to file for its oral multiple sclerosis drug fenebrutinib after a third phase 3 win, but could liver toxicity be a stumbling block?
R&D
Rare disease at an inflection point: Why the next wave will ...
As rare disease becomes more competitive, information parity is disappearing.
News
New biotech Atrium born out of Novartis/Avidity marriage
Atrium Therapeutics has launched with $270m in funding and two preclinical siRNA candidates for rare genetic cardiomyopathies.
R&D
Net Treatment Benefit in rare disease trials: Aligning trial...
Net Treatment Benefit provides a formal framework to integrate multiple prioritised outcomes into a single measure of overall treatment effect.
News
US lawmakers take FDA to task over rare diseases record
Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.
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