Article
Therapeutic Options for Mucopolysaccharidosis II (Hunter Disease).
Current pharmaceutical design - 1 Jan 2020
Kubaski Francyne, Vairo Filippo, Baldo Guilherme, de Oliveira Poswar Fabiano, Corte Amauri Dalla, Giugliani Roberto
Abstract excerpt
BACKGROUND: Mucopolysaccharidosis type II (Hunter syndrome, or MPS II) is an X-linked lysosomal disorder caused by the deficiency of iduronate-2-sulfatase, which leads to the accumulation of glycosaminoglycans (GAGs) in a variety of tissues, resulting in a multisystemic disease that can also impair the central nervous system (CNS). OBJECTIVE: This review focuses on providing the latest information and expert...
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