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UK patients get early access to Alnylam’s ultra-rare disease drug lumasiran
https://pharmaphorum.com/news/uk-patients-get-early-access-to-alnylams-ultra-rare-disease-drug-lumasiran/
Lojuxta will be available to patients this year
Strimvelis is most expensive drug cleared by NICE
A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy.
Managed access scheme will control costs to NHS.