News Amicus to file Fabry drug after FDA changes mind Regulator drops demand for further phase 3 trial.
News Sarepta announces Allergan veteran as next CEO Sarepta has announced former Allergan president Doug Ingram will be its new CEO.
News More good news for Bluebird Bio with CALD gene therapy Bluebird Bio has presented encouraging data from its gene therapy treatment for t
News Ionis claims first approval for Alexander disease therapy Ionis' Zanvastro has been approved by the FDA for Alexander disease, becoming the first disease-modifying therapy for the devastating rare disease.
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