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 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.
News EU clears first drug therapy for rare disease cALD Neuraxpharm and Minoryx' Nezglyal has become the first approved drug therapy in the EU for rare neurodegenerative disease cALD.
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