A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II
Work
Year: 2015
Type: article
Abstract: PurposeApproximately two-thirds of patients with the lysosomal storage disease mucopolysaccharidosis II have progressive cognitive impairment. Intravenous (i.v.) enzyme replacement therapy does not af... more
Source: Genetics in Medicine
Authors Joseph Muenzer, Christian J. Hendriksz, Zheng Fan, Suresh Vijayaraghavan, V. Hugh Perry +7 more
Institutions University of North Carolina at Chapel Hill, Boston Children's Hospital, Takeda (United States)
Cites: 17
Cited by: 159
Related to: 10
FWCI: 13.08
Citation percentile (by year/subfield): 100
Subfield: Physiology
Field: Medicine
Domain: Health Sciences
Sustainable Development Goal Good health and well-being
Open Access status: hybrid
APC paid (est): $4,200