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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/
ultra-rare diseases
NHS England okays funding for cholesterol-lowering drug
Lojuxta will be available to patients this year
NHS should fund GSK’s £526,000 “bubble baby” drug – NICE
Strimvelis is most expensive drug cleared by NICE
Orchard’s gene therapy gains UK ‘promising innovative medicine’ status
A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy.
Alexion price cut opens up Strensiq to more patients
Managed access scheme will control costs to NHS.
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