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Retricted access

Research article

First published 2012-12

Niemann-Pick Disease Type C: Implications for Sedation and Anesthesia for Diagnostic Procedures

Ning Miao, Xiaowei Lu, Naomi P. O’Grady [...]

Journal of Child Neurology

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Other

First published 2010-03

Long-Term Miglustat Therapy in Children With Niemann-Pick Disease Type C

Marc C. Patterson, Darleen Vecchio, Elizabeth Jacklin [...]

Journal of Child Neurology

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Research article

First published 2013-12

Effects of Miglustat on Stabilization of Neurological Disorder in Niemann–Pick Disease Type C

Parvaneh Karimzadeh, Seyed Hassan Tonekaboni, Mahmoud Reza Ashrafi [...]

Journal of Child Neurology

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Case report

First published 2015-10

Systemic lupus erythematosus occurring in a patient with Niemann–Pick type B disease

G Murgia, D Firinu, R Meleddu [...]

Lupus

Open access

Case report

First published 2024-7

Bilateral Macular Halo and Full-Thickness Macular Hole Repair in Niemann-Pick Disease Type B

Ye Li, Svetlana Cherepanoff, Adrian T. Fung

Journal of VitreoRetinal Diseases

Free access

Research article

First published 2014-9

Niemann–Pick Disease Type C

Daozhan Yu, Manju Swaroop, Mengqiao Wang [...]

Journal of Biomolecular Screening

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Research article

First published 2008-6

Type a Niemann-Pick Disease

A. D'Amico, M. Sibilio, F. Caranci [...]

The Neuroradiology Journal

Free access

Review article

First published 2016-5

Diagnostic workup and management of patients with suspected Niemann-Pick type C disease

Apostolos Papandreou, Paul Gissen

Therapeutic Advances in Neurological Disorders

Open access

Case report

First published 2022

Lamellar Bodies in Podocytes Associated With Compound Heterozygous Mutations for Niemann Pick Type C1 Mimicking Fabry Disease,a Case Report

Pairach Pintavorn, Stephanie Munie, Sweta Munagapati

Canadian Journal of Kidney Health and Disease

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Research article

First published 2026-4

An Analysis of Biomarkers for the Evaluation of Gene Therapy in Niemann–Pick Disease Type C1 Mice

Chika Watanabe, Masamitsu Maekawa, Eriko Jimbo [...]

Human Gene Therapy

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