Rare diseases

Sarepta goes up, PTC goes down – DMD research sways the market

https://pharmaphorum.com/news/dmd-therapy-research-sways-market/

Patients call for end to two-year row over CF drug funding

https://pharmaphorum.com/news/patients-call-for-end-to-two-year-row-over-cf-drug-funding/

Orphan drugs dominate 2018 UK Prix Galien shortlist

https://pharmaphorum.com/news/orphan-drugs-uk-prix-galien-shortlist/

Scotland’s NHS to fund Ipsen cancer diarrhoea drug

https://pharmaphorum.com/news/scotlands-nhs-ipsen-cancer-drug/

Official Agenda released for 8th annual Orphan Drugs and Rare Diseases conference

https://pharmaphorum.com/partner-content/official-agenda-released-for-8th-annual-orphan-drugs-and-rare-diseases-conference/

US lawmakers back ‘right to try’ legislation

https://pharmaphorum.com/news/us-lawmakers-back-right-to-try-legislation/

Spark/Pfizer eye phase 3 trial of haemophilia B gene therapy

https://pharmaphorum.com/news/spark-pfizers-haemophilia-b-drug-dramatically-reduces-bleeds/

Roche haemophilia data piles pressure on Shire

https://pharmaphorum.com/news/roche-haemophilia-data-piles-pressure-on-shire/

https://pharmaphorum.com/partner-content/open-health-invest-in-rare-disease/

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