Sage Journals: Discover world-class research

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

Retricted access

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

Open access

Research article

First published 2010-5

Chondroitin sulfate and growth factor signaling in the skeleton: Possible links to MPS VI

Tamara Alliston

Journal of Pediatric Rehabilitation Medicine

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

Open access

Research article

First published 2018

Long-Term Galsulfase Treatment Associated With Improved Survival of Patients With Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome)

Adrian Quartel, Paul R. Harmatz, Christina Lampe [...]

Journal of Inborn Errors of Metabolism and Screening

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

Free access

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

Retricted access

Research article

First published 1989-10

Transcultural Understanding of a Hereditary Disorder

Lauren Handelman, Samuel Menahem, I. Maurice Eisenbruch

Clinical Pediatrics

Free access

Research article

First published 2011-7

Thrombocytopenia associated with galsulfase treatment

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

Human & Experimental Toxicology

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

Search results