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Open access

Research article

First published 2013-02

Gene Therapy for Mucopolysaccharidosis Type VI Is Effective in Cats Without Pre-Existing Immunity to AAV8

Rita Ferla, Thomas O'Malley, Roberto Calcedo [...]

Human Gene Therapy

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Research article

First published 2017-01

Novel Mutations,Including a Large Deletion in the ARSB Gene,Causing Mucopolysaccharidosis Type VI

Chupong Ittiwut, Sukanya Boonbuamas, Chalurmpon Srichomthong [...]

Genetic Testing and Molecular Biomarkers

Free access

Research article

First published 2011-7

Thrombocytopenia associated with galsulfase treatment

Murat Doğan, Yasar Cesur, Erdal Peker [...]

Human & Experimental Toxicology

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Research article

First published 1999-03

Regulation of N-Acetylgalactosamine 4-Sulfatase Expression in Retrovirus-Transduced Feline Mucopolysaccharidosis Type VI Muscle Cells

Gouri Yogalingam, Vivienne Muller, John J. Hopwood [...]

DNA and Cell Biology

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Research article

First published 1997-10

Feline Mucopolysaccharidosis Type VI: Correction of Glycosaminoglycan Storage in Myoblasts by Retrovirus-Mediated Transfer of the Feline N-Acetylgalactosamine 4-Sulfatase Gene

Gouri Yogalingam, Julie Bielicki, John J. Hopwood [...]

DNA and Cell Biology

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Research article

First published 2010-02

Large Deletion Involving Exon 5 of the Arylsulfatase B Gene Caused Apparent Homozygosity in a Mucopolysaccharidosis Type VI Patient

Guglielmo R.D. Villani, Michela Grosso, Gianfranco Pontarelli [...]

Genetic Testing and Molecular Biomarkers

Open access

Research article

First published 2017

Impact of Enzyme Replacement Therapy in a Patient Younger Than 2 Years Diagnosed With Maroteaux-Lamy Syndrome (MPS VI)

Johanna Acosta Guio, Gustavo AdolfoGiraldo-Ospina

Journal of Inborn Errors of Metabolism and Screening

Free access

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

Free access

Research article

First published 2018-3

Mucopolysaccharidosis Type VI in a Great Dane Caused by a Nonsense Mutation in the ARSB Gene

Ping Wang, Carol Margolis, Gloria Lin [...]

Veterinary Pathology

Open access

Case report

First published 2023

Successful combined umbilical cord blood and bone marrow transplantation from an HLA-matched sibling for MPS VI: a case report

Pankti Haria, Vinayak Kedage, Pradnya Dalvi [...]

Therapeutic Advances in Rare Disease

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