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.
R&D
How innovative trial approaches are advancing rare disease r...
Innovative trial designs that harness data augmentation, RWD, and the power of AI are enabling a new era of rare disease research.
News
Generate's IPO gets over the line, raising $400m
AI-powered drug developer Generate Biomedicine completes a $400m IPO, the largest for biotech on the Nasdaq so far this year.
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