Early initiation of enzyme replacement therapy for the mucopolysaccharidoses

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Enzyme replacement therapy for Gaucher's disease: the early Canadian experience.

BACKGROUND The management of severe Gaucher's disease was dramatically improved by the development of enzyme replacement therapy. However, this treatment is very costly (currently about $21,000 per infusion for adults at the starting dose recommended by the manufacturer). The goal of this study was to determine how enzyme replacement therapy was being prescribed and financially supported in var...

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Therapy for the mucopolysaccharidoses.

Better understanding of disease pathophysiology, improved supportive care and availability of disease-specific treatments for some of the mucopolysaccharidosis (MPS) disorders have greatly improved the outlook for patients with MPS disorders. Optimal management of these multisystemic disorders involves a multidisciplinary team and regular, comprehensive follow-up. Enzyme replacement therapy (ER...

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Too early initiation of renal replacement therapy may be harmful

In an observational multicenter study, Elseviers and colleagues report that renal replacement therapy (RRT) in acutely ill patients treated for acute kidney injury is an independent risk factor for death. This result may question the benefit of the current practice of early RRT initiation.

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ژورنال

عنوان ژورنال: Molecular Genetics and Metabolism

سال: 2014

ISSN: 1096-7192

DOI: 10.1016/j.ymgme.2013.11.015