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Ionis’ FCS drug approved in EU, launch due in Germany
https://pharmaphorum.com/news/ionis-fcs-drug-approved-in-eu-launch-due-in-germany/
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.
Sucampo buys rare disease firm Vtesse
Sucampo gains rights to a drug for rare genetic disorder.
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