Views & Analysis Meeting the challenges of rare disease treatment adherence HCPs, pharma and patients can pool their experience to develop effective, tailored support.
News Europe delays review of PTC's Duchenne drug Review of marketing authorisation delayed, new trial likely.
News How will Brexit affect rare disease research? The decision to leave the European Union is causing concern across all healthcare disciplines, with few answers to the many questions about where the UK will stand in relation to Europe.
News Merck rare tumour drug Ezmekly backed for NHS use Merck's drug for tumours associated with rare disease neurofibromatosis type 1, Ezmekly, has been cleared for NHS use in children as young as two.
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