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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

Free access

Research article

First published 2021-1

Temporospatial Development of Neuropathologic Findings in a Canine Model of Mucopolysaccharidosis IIIB

Tyler A. Harm, Shannon J. Hostetter, Ariel S. Nenninger [...]

Veterinary Pathology

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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Research article

First published 2015-12

A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB

Aaron S. Meadows, F. Jason Duncan, Marybeth Camboni [...]

Human Gene Therapy Clinical Development

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Research article

First published 2014-06

Feasibility and Safety of Systemic rAAV9-h NAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology,Biodistribution,and Immunological Assessments in Primates

Darren A. Murrey, Bartholomew J. Naughton, F. Jason Duncan [...]

Human Gene Therapy Clinical Development

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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

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Research article

First published 2026-3

Distinct Brain Magnetic Resonance Imaging (MRI) Findings Across Mucopolysaccharidosis Types: Novel Insights

Nisa Baspinar, Havva Yazici, Cenk Eraslan [...]

Journal of Child Neurology

Open access

Research article

First published 2016

The Link Between Lysosomal Storage Disorders and More Common Diseases

Michael Beck

Journal of Inborn Errors of Metabolism and Screening

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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

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