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 NHS "isn't delivering equitable care for rare diseases" A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.
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 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.
Market Access What rare disease commercialisation in Europe teaches us - a... Rare disease launches are the ultimate stress test for European commercialisation.
News Non-profit DNDi preps phase 3 trials for dengue antibody Non-profit DNDi has licensed a dengue therapy from India's SII, paving the way for a phase 3 trial in Malaysia, Thailand, and Brazil.
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