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NICE rejects Alnylam and Akcea’s amyloidosis drugs
https://pharmaphorum.com/news/nice-rejects-alnylam-and-akceas-amyloidosis-drugs/
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.
Alexion appoints former Baxalta chief Hantson as CEO
Former AZ chief David Brennan expected to take over as chairman in May.
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