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Tags: Lysosomal Storage Disorders

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Mucopolysaccharidosis type I, II and VI and response to enzyme replacement therapy: Results from a single-center case series study

Mucopolysaccharidoses (MPS) types I, II and VI are associated with deficiencies in alpha-L-iduronidase, iduronate-2-sulfatase and N-acetylgalactosamine-4-sulfatase, respectively, and generally involve progressive and multi-systemic clinical…

Tags: 2017, Agarwal A, Albano LMJ, El Dib R, Franco Jfds, Glycosaminoglycans, Intractable Rare Dis Res, Kim CA, Lysosomal Storage Disorders, Milhan NVM, November 2017 List, Prognosis, Soares D, Treatment

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