News AZ, Ionis build case for amyloidosis drug eplontersen Long-term data in ATTR polyneuropathy boost drug's credentials ahead of December FDA decision.
News Pharming brings first drug for rare disease APDS to US Joenja will launch in April and could make upwards of $200m a year in sales, say analysts.
News PTC’s gene therapy for rare disease cleared for NHS use Upstaza has shown impressive clinical results in children with fatal disorder AADC deficiency.
Digital AI technology: A game-changer for rare disease diagnosis AI technology isn’t designed to replace clinicians - its function is to help them.
News FDA sets new decision date for Ipsen’s FOP therapy US agency sets 23rd August deadline for palovarotene review as Ipsen files more clinical data.
Digital Leave no one behind: Digital user research in rare disease Digital solutions have the potential to bridge care gaps for rare disease patients.
News Novo sues Lilly over 'misleading' GLP-1 ad campaigning The rivalry between Novo Nordisk and Eli Lilly in the GLP-1 market has spilled over into the courts in the US, with claims of misleading advertising.
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