Mucopolysaccharidosis Type II (Hunter Syndrome): Clinical Picture and Treatment
Until recently, no specific treatment was available for the affected patients; management mainly consisted of supportive care and treatment of complications. Enzyme replacement therapy with recombinant iduronate-2-sulphatase (idursulfase), however, has now been introduced. And it could be demonstrated that weekly intravenous infusions of idursulfase is able to improve many of the symptoms and signs of Hunter syndrome. This review will present the efficacy and safety data of the enzyme preparation and discuss benefits and limitations of this new therapeutic option.
Keywords: Enzyme replacement therapy; Hunter syndrome; X-linked lysosomal storage disorder; efficacy; haematopoietic stem cell therapy; intravenous infusions of idursulfase; lysosomal enzyme iduronate-2-sulfatase (IDS); mucopolysaccharidosis type II; obstructive and restrictive airway disease; retinal dysfunction; substrate optimization; substrate reduction; treatment; valvular dysfunction
Document Type: Research Article
Publication date: June 1, 2011
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