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

Retricted access

Research article

First published 2017-12

Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy

Haiyan Fu, Aaron S. Meadows, Ricardo J. Pineda [...]

Human Gene Therapy Clinical Development

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

Open access

Research article

First published 2018

Understanding the Early Presentation of Mucopolysaccharidoses Disorders

Lorne Clarke, Carolyn Ellaway, Helen E. Foster [...]

Journal of Inborn Errors of Metabolism and Screening

Retricted access

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

Research article

First published 2014-8

Hyperactivity,Unexplained Speech Delay,and Coarse Facies—Is It Sanfilippo Syndrome?

Arushi Gahlot Saini, Pratibha Singhi, Jitendra Kumar Sahu [...]

Journal of Child Neurology

Retricted access

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

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

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)

Retricted access

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

Search results