Sage Journals: Discover world-class research

Retricted access

Review article

First published 2019-10

In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon

Sara Marcó, Virginia Haurigot, Fatima Bosch

Human Gene Therapy

Open access

Case report

First published 2024

Successful use of an eye gaze AAC communication board by a young adult with advanced Sanfilippo Syndrome (MPS IIIA): Case report

Christine Brennan, Abigail Matthews, Sherri Tennant

Therapeutic Advances in Rare Disease

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Other

First published 2015-5

A Clinical Multicenter Study of Orofacial Features in 26 Brazilian Patients with Different Types of Mucopolysaccharidosis

Erlane Marques Ribeiro, Cristiane S.R. Fonteles, Adriana Bezerra Freitas [...]

The Cleft Palate Craniofacial Journal

Retricted access

Case report

First published 2025-7

Rare Presentation of Attenuated Mucopolysaccharidosis Type IIIA as Isolated Retinitis Pigmentosa

Josephine R. Seela, Jade Y. Moon, Sandra R. Montezuma

Journal of VitreoRetinal Diseases

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Other

First published 2010-10

Hemiatrophy of the Spinal Cord in a Patient With Mucopolysaccharidosis Type IIIB

Pauline Samia, Nicky Wieselthaler, George F. van der Watt [...]

Journal of Child Neurology

Open access

Research article

First published 2026-5

Carpal tunnel syndrome in mucopolysaccharidosis type I: clinical,surgical and histopathological findings

Boudewijn AW van Binsbergen, Joris A van Dongen, Linda Vriend [...]

Journal of Hand Surgery (European Volume)

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Research article

First published 2017-2

Natural History of Sanfilippo Syndrome Type C in Boyacá,Colombia

Harvy Mauricio Velasco, Yasmin Sanchez, Angela Milena Martin [...]

Journal of Child Neurology

Open access

Research article

First published 2018

Outcomes of a Physician Survey on the Type,Progression,Assessment,and Treatment of Neurological Disease in Mucopolysaccharidoses

Maurizio Scarpa, Paul R. Harmatz, Bianca Meesen [...]

Journal of Inborn Errors of Metabolism and Screening

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

Free access

Research article

First published 2015-1

Pathological and Biochemical Studies of Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B) in Juvenile Emus ( Dromaius novaehollandiae )

C. Palmieri, U. Giger, P. Wang [...]

Veterinary Pathology

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