News Kanuma approval adds to Alexion rare disease roster Alexion has gained FDA approval for Kanuma, its treatment for the rare disease lysosomal acid lipase (LAL) deficiency.
Articles Rare disease drug Vimizim gets NICE 'yes' – but needs real-w... NICE says it can approve BioMarin's ultra-rare drug treatment Vimizim, but has proposed real-world data collection and a price cap to overcome doubts about the drug's value, reports Andrew McCo
Articles A history of Shire The history of one of the world's biggest speciality and rare disease pharmaceutical companies
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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