Discovery and clinical development of idursulfase (Elaprase®) for the treatment of mucopolysaccharidosis II (Hunter syndrome)

M Heartlein, A Kimura - 2014 - books.rsc.org
Heparan sulfate (HS) and dermatan sulfate (DS) are glycosaminoglycans (GAGs) comprised
of linear sulfated chains of alternating uronic acid and hexosamine residues that are modi …

Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future

DAH Whiteman, A Kimura - Drug design, development and therapy, 2017 - Taylor & Francis
Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare,
multisystemic, progressive lysosomal storage disease caused by deficient activity of the …

Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II

AR Garcia, JM DaCosta, J Pan, J Muenzer… - Molecular genetics and …, 2007 - Elsevier
Mucopolysaccharidosis II (MPS II; Hunter syndrome) is an X-linked metabolic disorder
caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S), which …

Review of the use of idursulfase in the treatment of mucopolysaccharidosis II

TA Burrow, ND Leslie - Biologics: Targets and Therapy, 2008 - Taylor & Francis
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a heterogeneous, progressive X-
linked recessively inherited lysosomal storage disease that is caused by a deficiency of the …

Mucopolysaccharidosis type II (Hunter syndrome): Clinical and biochemical aspects of the disease and approaches to its diagnosis and treatment

S Mohamed, QQ He, AA Singh, V Ferro - Advances in Carbohydrate …, 2020 - Elsevier
Abstract Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a rare X-linked
lysosomal storage disease caused by mutations of the gene encoding the lysosomal …

[HTML][HTML] Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series

MY Chan, AJ Nelson, LH Ngu - Molecular Genetics and Metabolism …, 2023 - Elsevier
Abstract Mucopolysaccharidosis (MPS) type II (Hunter syndrome) is a rare X-linked,
recessive, lysosomal storage disorder caused by the deficit of the enzyme iduronate 2 …

Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)

EMK Da Silva, MWL Strufaldi… - Cochrane Database …, 2011 - cochranelibrary.com
Background Mucopolysaccharidosis II, also known as Hunter syndrome, is a rare, X‐linked
disease caused by a deficiency of the lysosomal enzyme iduronate‐2‐sulfatase, which …

Idursulfase in Hunter syndrome treatment.

G Zareba - Drugs of today (Barcelona, Spain: 1998), 2007 - europepmc.org
Hunter syndrome (mucopolysaccharidosis II, MPS II) is a rare X-linked lysosomal storage
disorder caused by the deficiency of enzyme iduronate-2-sulfatase (I2S), which results in …

A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome)

J Muenzer, M Gucsavas-Calikoglu… - Molecular genetics and …, 2007 - Elsevier
OBJECTIVE: To evaluate the safety and explore the efficacy of idursulfase (recombinant
human iduronate-2-sulfatase) treatment for mucopolysaccharidosis II (MPS II). STUDY …

Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy

SC Jung, ES Park, EN Choi, CH Kim, SJ Kim, DK Jin - Molecules and cells, 2010 - Springer
Abstract Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked inherited
disorder caused by a deficiency of the enzyme iduronate-2-sulfatase (IDS), which results in …