News Norgine gets first EU approval for WHIM syndrome therapy Patients in the EU with the ultra-rare disease WHIM syndrome now have their first approved treatment, Norgine's Xolremdi.
News Chiesi signs $1.9bn deal to buy KalVista and its HAE drug Chiesi has boosted its rare disease business with an agreement to buy KalVista and Ekterly, its oral drug for treating attacks in rare disease HAE.
News Intellia files CRISPR drug after historic phase 3 readout Intellia has reported the first-ever phase 3 results with an in vivo gene-editing therapy, CRISPR-based lonvo-z for hereditary angioedema.
News Novo Nordisk preps filings for sickle cell drug etavopivat Novo Nordisk has claimed a key win in its head-to-head race with Agios to bring an oral PKR activator to the market for sickle cell disease.
News Italy leads the way on child genetic disease diagnosis A scheme to hasten the diagnosis of children with genetic disorders is achieving strong results and could be a template for national health systems.
News Travere claims first FDA OK in rare kidney disease FSGS Shrugging off an earlier phase 3 disappointment, Travere’s Filspari has become the first FDA-approved treatment for rare kidney disease FSGS.
News GLP-1s drive Lilly's Q1, but it's reticent on oral launch Investors are itching for news on the rollout of Lilly's oral weight-loss drug Foundayo, but there's little information in its first-quarter update.
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