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Promise of CF combinations lifts Vertex, reignites takeover interest
https://pharmaphorum.com/news/vertex-hopes-pipeline-combos-will-expand-cf-offering/
Rare diseases
Amicus to file Fabry drug after FDA changes mind
July 12, 2017Regulator drops demand for further phase 3 trial.
EMA and FDA aim to slash Gaucher disease R&D costs
July 5, 2017Proposals include multi-company trial.
Sarepta announces Allergan veteran as next CEO
July 3, 2017
NICE recommends Sanofi’s Gaucher treatment
June 28, 2017Cancer drugs from Novartis and Pfizer also backed
More good news for Bluebird Bio with CALD gene therapy
June 27, 2017
UK patients gain early access to Duchenne drug
June 26, 2017Patients to get Santhera’s Raxone under EAMS scheme.
Shire long-acting ADHD drug finally approved
June 21, 2017Mydadis approval ends decade-long saga with FDA.
