ultra-rare diseases

NHS England okays funding for cholesterol-lowering drug

https://pharmaphorum.com/news/nhs-england-okays-funding-for-cholesterol-lowering-drug-lojuxta/

NHS should fund GSK’s £526,000 “bubble baby” drug – NICE

https://pharmaphorum.com/news/nhs-fund-gsks-526000-bubble-baby-drug-nice/

GSK’s ‘bubble baby’ gene therapy passes NICE’s new stricter test

https://pharmaphorum.com/news/strimvelis-approved-by-nice/

Orchard’s gene therapy gains UK ‘promising innovative medicine’ status

https://pharmaphorum.com/news/orchards-gene-therapy-gains-uk-promising-innovative-medicine-status/

Alexion price cut opens up Strensiq to more patients

https://pharmaphorum.com/news/alexions-strensiq-available-patients-nice-rethink/

Outrage over Duchenne drug price reinflamed by PTC’s $35,000 price tag

https://pharmaphorum.com/news/dmd-families-will-still-pay-huge-premium-old-drug-says-ptc/

Sucampo buys rare disease firm Vtesse

https://pharmaphorum.com/news/sucampo-buys-rare-disease-firm-vtesse/

Alexion appoints former Baxalta chief Hantson as CEO

https://pharmaphorum.com/news/alexion-appoints-former-baxalta-chief-hantson-ceo/

NICE rejects second Alexion ultra-rare disease drug

https://pharmaphorum.com/news/alexion-furious-nice-rejects-ultra-rare-disease-drug/

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