Enzyme replacement therapy for the management of the mucopolysaccharidoses.
Enzyme replacement therapy (ERT) is now available for several of the mucopolysaccharidosis disorders. This brief review summarizes the role of ERT in reducing the burden of peripheral disease in many patients with mucopolysaccharidosis disorders, and describes the challenges that remain in treating the neurological manifestations of these conditions.
Wraith
J E
JE
Willink Biochemical Genetics Unit, Royal Manchester Children's Hospital, Manchester, UK. ed.wraith@cmmc.nhs.uk
D003131:Combined Modality Therapy / Q000379:methods; D056947:Enzyme Replacement Therapy / Q000379:methods*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D007066:Iduronate Sulfatase / Q000627:therapeutic use; D007068:Iduronidase / Q000627:therapeutic use; D008059:Mucopolysaccharidosis I / Q000628:therapy*; D016532:Mucopolysaccharidosis II / Q000628:therapy*; D009087:Mucopolysaccharidosis VI / Q000628:therapy*; D020051:N-Acetylgalactosamine-4-Sulfatase / Q000627:therapeutic use; D011994:Recombinant Proteins / Q000627:therapeutic use
D011994:Recombinant Proteins; D020051:N-Acetylgalactosamine-4-Sulfatase; C508864:galsulfase; D007066:Iduronate Sulfatase; D007068:Iduronidase