News Alexion lambasts NICE over rare disease ruling Watchdog accused of ignoring stakeholder consensus and call for negotiations
News Rare disease drug Strensiq too expensive, says NICE Alexion's new rare disease treatment Strensiq has been rejected by England's health watchdog NICE in draft guidance.
News Neurogene drops high-dose arm in Rett study after death The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died
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