BioMarin gene therapy for haemophilia on course for EU approval
BioMarin, EU, gene therapy, haemophilia A, regulatory approval, Roctavian
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BioMarin
NICE firms up NHS use of Vimizim for Morquio A syndrome
BioMarin, HEOR, Morquio, NHS England, NICE, rare disease, Vimizim
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FDA slaps clinical hold on BioMarin’s PKU gene therapy
BioMarin, fda, gene therapy, phenylketonuria, rare disease, safety
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2nd Gene Therapy for Blood Disorders
BioMarin, blood disorders, gene therapy, Hemophilia, sickle cell disease
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Gene Therapy for Blood Disorders Digital Summit
Girl loses High Court battle over NICE review of BioMarin’s PKU drug
BioMarin, Highly Specialised Technology, NICE, phenylketonuria, rare disease
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Child with rare disease takes NICE to court over access to life-changing therapy
BioMarin, cost-effectiveness, NICE, phenylketonuria, Rare diseases
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BioMarin seeks haemophilia gene therapy approval after promising data
BioMarin, gene therapy, haemophilia, valoctocogene roxaparvovec
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