Rare diseases Archives - Pharmaphorum

UK biotech Orchard plans $172.5m US IPO to fund rare disease R&D

https://pharmaphorum.com/news/uk-biotech-orchard-plans-172-5m-us-ipo-to-fund-rare-disease-rd/

Preliminary Attendee List Released for Orphan Drugs & Rare Diseases Conference

https://pharmaphorum.com/partner-content/preliminary-attendee-list-released-for-orphan-drugs-rare-diseases-conference/

FDA approves Ionis/Akcea’s Tegsedi RNAi drug, taking on Alnylam

https://pharmaphorum.com/news/fda-approves-ionis-akceas-tegsedi-competing-against-alnylam/

Roche’s Hemlibra approved in expanded haemophilia A use

https://pharmaphorum.com/news/roches-hemlibra-approved-in-expanded-haemophilia-a-use/

Denmark funds Vertex’s CF drug Orkambi, UK talks deadlocked

https://pharmaphorum.com/news/denmark-funds-vertexs-cf-drug-orkambi-uk-talks-deadlocked/

Alnylam rare disease drug may be delayed

https://pharmaphorum.com/news/alnylam-rare-disease-drug-may-be-delayed/

Alexion buys rare diseases biotech Syntimmune for up to $1.2bn

https://pharmaphorum.com/news/alexion-buys-rare-diseases-biotech-syntimmune-for-up-to-1-2bn/

CSL Behring lung drug would cost NHS £8m per patient, says NICE

https://pharmaphorum.com/news/csl-behring-lung-drug-would-cost-nhs-8m-per-patient-says-nice/

Alexion prepares filings for new rare disease use for Soliris

https://pharmaphorum.com/news/alexion-prepares-filings-for-new-rare-disease-use-for-soliris/

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