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The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

Harmatz, P.; Lampe, C.; McGill, J.J.; Parini, R.; Leao-Teles, E.; Valayannopoulos, V.; Cole, T.J.; Matousek, R.; Graham, S.; Guffon, N.; Quartel, A. (Elsevier, 2017-09)

Mucopolysaccharidosis (MPS) VI is an autosomal recessive lysosomal storage disorder arising from deficient activity of N-acetylgalactosamine-4-sulfatase (arylsulfatase B) and subsequent intracellular accumulation of ...

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