Rare diseases

Pfizer axes development of Duchenne muscular dystrophy antibody

https://pharmaphorum.com/news/duchenne-trials-axed-pfizer-dmd/

US regulators reject Akcea and Ionis rare disease drug

https://pharmaphorum.com/news/us-regulators-reject-akcea-ionis-rare-disease-drug/

Neurofibromatosis treatment granted orphan status by EMA

https://pharmaphorum.com/news/nf1-treatment-granted-orphan-status-ema/

The first lipodystrophy treatment for European patients

https://pharmaphorum.com/news/the-first-lipodystrophy-treatment-for-european-patients/

Shionogi’s Mulpleta gets FDA green light for patients with liver disease

https://pharmaphorum.com/news/shionogis-mulpleta-gets-fda-green-light-for-patients-with-liver-disease/

Alexion hastens review of successor to rare disease blockbuster Soliris

https://pharmaphorum.com/news/alexion-hastens-review-of-successor-to-rare-disease-blockbuster-soliris/

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/

Scotland’s NHS to fund Ipsen cancer diarrhoea drug

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

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

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

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