Rare diseases

FCS: A life-limiting condition

https://pharmaphorum.com/views-analysis-patients/fcs-a-life-limiting-condition/

Ipsen adds Blueprint’s FOP rare disease drug to pipeline for $535m

https://pharmaphorum.com/news/ipsen-adds-blueprints-fop-rare-disease-drug-to-pipeline-for-535m/

UCB sets sights on rare diseases giant Alexion with Ra Pharma acquisition

https://pharmaphorum.com/news/ucb-takes-on-rare-diseases-giant-alexion-with-ra-pharma-acquisition/

Rare disease patients unconvinced by health secretary’s post-Brexit research plans

https://pharmaphorum.com/news/rare-disease-patients-unconvinced-by-health-secretarys-post-brexit-rd-plans/

European regulators grant fast path to market for Calliditas’ rare kidney disease drug

https://pharmaphorum.com/news/european-grant-fast-path-to-market-for-calliditas-rare-kidney-disease-drug/

AveXis rules out EU compassionate use of SMA gene therapy, says Biogen option is available

https://pharmaphorum.com/news/avexis-rules-out-eu-compassionate-use-of-sma-gene-therapy-zolgensma/

Exclusive Bionical EMAS speaker interview released for Orphan Drugs & Rare Diseases

https://pharmaphorum.com/partner-content/exclusive-bionical-emas-speaker-interview-released-for-orphan-drugs-rare-diseases/

Brexit could freeze UK out of EU rare disease research, says patient group

https://pharmaphorum.com/news/brexit-could-freeze-uk-out-of-eu-rare-disease-research-says-patient-group/

bluebird presents long-term data from CALD gene therapy

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

1 2 3 4 5 >»

Close in 10 seconds

Pharmaphorum.com

Search