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New FDA knockback hits uniQure and Huntington patients hard
uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, delaying the highly anticipated programme.
News
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.
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