A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II | Zendy

Joseph Muenzer | Zendy; Christian J. Hendriksz | Zendy; Zheng Fan | Zendy; Suresh Vijayaraghavan | Zendy; V. Hugh Perry | Zendy; Saikat Santra | Zendy; Guirish A. Solanki | Zendy; Mary Ann Mascelli | Zendy; Luying Pan | Zendy; Nan Wang | Zendy; Kenneth Sciarappa | Zendy; Ann Barbier | Zendy

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A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

Author(s) -

Joseph Muenzer,

Christian J. Hendriksz,

Zheng Fan,

Suresh Vijayaraghavan,

V. Hugh Perry,

Saikat Santra,

Guirish A. Solanki,

Mary Ann Mascelli,

Luying Pan,

Nan Wang,

Kenneth Sciarappa,

Ann Barbier

Publication year - 2015

Publication title -

genetics in medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 3.509

H-Index - 128

eISSN - 1530-0366

pISSN - 1098-3600

DOI - 10.1038/gim.2015.36

Subject(s) - hunter syndrome , mucopolysaccharidosis type ii , enzyme replacement therapy , medicine , mucopolysaccharidosis , clinical endpoint , cerebrospinal fluid , adverse effect , anesthesia , surgery , randomized controlled trial , disease

Approximately two-thirds of patients with the lysosomal storage disease mucopolysaccharidosis II have progressive cognitive impairment. Intravenous (i.v.) enzyme replacement therapy does not affect cognitive impairment because recombinant iduronate-2-sulfatase (idursulfase) does not penetrate the blood-brain barrier at therapeutic concentrations. We examined the safety of idursulfase formulated for intrathecal administration (idursulfase-IT) via intrathecal drug delivery device (IDDD). A secondary endpoint was change in concentration of glycosaminoglycans in cerebrospinal fluid.

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