Rare diseases Archives - Pharmaphorum

bluebird presents long-term data from CALD gene therapy

https://pharmaphorum.com/news/bluebird-presents-long-term-data-from-cald-gene-therapy/

HAE patients get new option as NICE backs Takeda’s Takhzyro

https://pharmaphorum.com/news/hae-patients-get-new-option-as-nice-backs-takedas-takhzyro/

Acceleron axes clinical development of muscular dystrophy drug

https://pharmaphorum.com/news/acceleron-axes-clinical-development-of-muscular-dystrophy-drug/

Orchard to file gene therapy in life-threatening rare disease next year

https://pharmaphorum.com/news/orchard-to-file-gene-therapy-in-life-threatening-rare-disease-next-year/

https://pharmaphorum.com/news/nhs-funds-eye-gene-therapy-after-novartis-cuts-613000-price/

Mike Page, Alexion Pharmaceuticals invite to SMi’s Orphan Drugs & Rare Diseases

https://pharmaphorum.com/partner-content/mike-page-alexion-pharmaceuticals-invite-to-smis-orphan-drugs-rare-diseases/

FDA agrees to downsized trial for Calliditas’ rare disease drug

https://pharmaphorum.com/news/fda-agrees-to-downsized-trial-for-calliditas-rare-disease-drug/

Alexion’s Soliris approved in new optic nerve disease use in Europe

https://pharmaphorum.com/news/alexions-soliris-approved-in-new-optic-nerve-disease-use-in-europe/

NHS reimburses Roche’s Hemlibra in new group of haemophilia patients

https://pharmaphorum.com/news/roche-hemlibra-haemophilia/

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