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?
News CHMP backs Ipsen drug for childhood brain cancer Ipsen's Ojemda is on course to become the first targeted medicine in the EU for some children with a form of brain cancer known as low-grade glioma.
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.
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.
News Moderna closes on EU approval for combined flu/COVID jab Moderna's combined vaccine for both flu and COVID-19 has been recommended for approval in the EU, after being knocked back in the US.
News Boehringer gets FDA's second 'national priority' approval Boehringer Ingelheim's lung cancer drug Hernexeos has become the second medicine approved after an FDA national priority review.
News Ipsen' Bylvay fluffs its lines in biliary atresia trial Ipsen's IBAT inhibitor Bylvay has failed a trial in biliary atresia, a rare and serious liver disease that affects babies and has limited treatments.
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