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Research article

First published 2010-2

Bone density assessment in patients with mucopolysaccharidosis: A preliminary report from patients with MPS II and VI

Ellen B. Fung, Jo Ann Johnson, Jacqueline Madden [...]

Journal of Pediatric Rehabilitation Medicine

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Research article

First published 2023-01

Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction

Kelly M. Podetz-Pedersen, Kanut Laoharawee, Sajya Singh [...]

Human Gene Therapy

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Research article

First published 2010-5

Musculoskeletal health in Hunter disease (MPS II): ERT improves functional outcomes

Klane K White, Susan Hale, Michael J Goldberg

Journal of Pediatric Rehabilitation Medicine

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Research article

First published 1999-9

Freezing Characteristics of Genetically Modified Lymphocytes for the Treatment of MPS II

A. Hubel, T. B. Darr, J. A. Norman

Cell Transplantation

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Research article

First published 2025-04

Central Nervous System-Targeted Gene Therapy for the Treatment of Neurocognitive Deficits in Mucopolysaccharidosis Type II Mice

Guoqing Chen, Xia Zhan, Xiaolan Gao [...]

Human Gene Therapy

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Research article

First published 2016-11

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice

Christian Hinderer, Nathan Katz, Jean-Pierre Louboutin [...]

Human Gene Therapy

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Research article

First published 2015-06

Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II

Taichi Wakabayashi, Yohta Shimada, Kazumasa Akiyama [...]

Human Gene Therapy

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Research article

First published 2014-5

Biomarkers of bone remodeling in children with mucopolysaccharidosis types I,II,and VI

David A. Stevenson, Kyle Rudser, Alicia Kunin-Batson [...]

Journal of Pediatric Rehabilitation Medicine

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Research article

First published 2017-08

Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed,AAV9-Mediated Iduronate Sulfatase Gene Transfer

Kanut Laoharawee, Kelly M. Podetz-Pedersen, Tam T. Nguyen [...]

Human Gene Therapy

Open access

Research article

First published 2015

A Cerebrospinal Fluid Collection Study in Pediatric and Adult Patients With Hunter Syndrome

Christian J. Hendriksz, Joseph Muenzer, Barbara K. Burton [...]

Journal of Inborn Errors of Metabolism and Screening

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