Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I
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چکیده
منابع مشابه
modern surgical techniques in treatment of patients with cleft lip & cleft palate
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15 صفحه اولLaronidase for treating mucopolysaccharidosis type I.
Mucopolysaccharidoses are a group of inherited metabolic diseases caused by the absence or deficiency of the lysosomal enzymes that are needed for breaking down glycosaminoglycans (GAGs). Over time, GAGs collect in cells, blood and connective tissues, and increased amounts are excreted in the urine. The result is permanent and includes progressive cell damage that affects the individual's appea...
متن کاملImmune tolerance induction for laronidase treatment in mucopolysaccharidosis I
Enzyme replacement therapy (ERT) can produce anti-drug antibody (ADA) responses that reduce efficacy or lead to hypersensitivity reactions. Six patients with severe mucopolysaccharidosis type I (MPS I/Hurler syndrome) who did not receive hematopoietic stem cell transplantation underwent an immunosuppression regimen prior to initiating ERT with laronidase. The primary endpoint for immune toleran...
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ژورنال
عنوان ژورنال: The Journal of Pediatrics
سال: 2016
ISSN: 0022-3476
DOI: 10.1016/j.jpeds.2016.08.033