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Retricted access

Research article

First published 2018-11

Multiple Sulfatase Deficiency: A Case Series With a Novel Mutation

Leen Hijazi, Amna Kashgari, Majid Alfadhel

Journal of Child Neurology

Retricted access

Research article

First published 1992-4

Saudi Variant of Multiple Sulfatase Deficiency

A. Al Aqeel, P.T. Ozand, J. Brismar [...]

Journal of Child Neurology

Retricted access

Research article

First published 1991-07

Multiple Sulfatase Deficiency With Early Severe Retinal Degeneration

Michael Harbord, J. Raymond Buncic, S.A. Chuang [...]

Journal of Child Neurology

Retricted access

Research article

First published 2025-11

Exploration Into Lived Experiences of Multiple Sulfatase Deficiency–Affected Individuals and Their Families

Francesco Gavazzi, Emily Yu, Zarrin Tashnim [...]

Journal of Child Neurology

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Other

First published 2004-06

Cerebral Glycolipidoses: Clinical Characteristics of 41 Pediatric Patients

Atchayaram Nalini, Rita Christopher

Journal of Child Neurology

Open access

Research article

First published 2010-5

Clinical utility of endurance measures for evaluation of treatment in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

Arline McDonald, Robert Steiner, Kerry Kuehl [...]

Journal of Pediatric Rehabilitation Medicine

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Research article

First published 2005-08

Coexpression of Formylglycine-Generating Enzyme Is Essential for Synthesis and Secretion of Functional Arylsulfatase A in a Mouse Model of Metachromatic Leukodystrophy

Yukiko Takakusaki, Sanae Hisayasu, Yukihiko Hirai [...]

Human Gene Therapy

Free access

Case report

First published 1998-5

Clinical and Morphologic Features of Mucopolysaccharidosis Type II in a Dog: Naturally Occurring Model of Hunter Syndrome

M. J. Wilkerson, D. C. Lewis, S. L. Marks [...]

Veterinary Pathology

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Research article

First published 2024-12

Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice

Angélica María Herreño-Pachón, Kazuki Sawamoto, Molly Stapleton [...]

Human Gene Therapy

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Research article

First published 2026-2

Intravenous Delivery of a Gene Therapy Vector that Expresses an Antitransferrin Receptor 1 Nanobody-I2S Fusion Protein Rescued Central Nervous System Lysosomal Burden in Mucopolysaccharidosis II Mice

Wanida Ruangsiriluk, Sarah Melissa P. Jacobo, Andrea Iskenderian [...]

Human Gene Therapy

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