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Open access

Research article

First published 2025

Visual Recovery and Neurological Stabilization Following Miglustat Treatment in Pediatric CLN3 Disease

Alice E. Dutton, Ineka T. Whiteman, Michael M. Jones [...]

Journal of Child Neurology

Open access

Review article

First published 2026

Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease

Robert J. Hopkin, Barry J. Byrne, Mazen M. Dimachkie [...]

Therapeutic Advances in Rare Disease

Retricted access

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

Retricted access

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

Retricted access

Research article

First published 2016-06

Characterization of the Zebrafish Homolog of β-Glucosidase 2: A Target of the Drug Miglustat

Saki Sultana, Nhu Y. Truong, Douglas B. Vieira [...]

Zebrafish

Open access

Brief report

First published 2026

Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials

Henning Andersen, Jordi Díaz-Manera, Ozlem Goker-Alpan [...]

Therapeutic Advances in Rare Disease

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

Retricted access

Research article

First published 2023-3

A Systematic Review and Meta-analyses of Longitudinal Studies on Drug Treatments for Gaucher Disease

Letícia Paula Leonart, Mariana M. Fachi, Beatriz Böger [...]

Annals of Pharmacotherapy

Free access

Research article

First published 2014-9

Niemann–Pick Disease Type C

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

Journal of Biomolecular Screening

Retricted access

Research article

First published 2013-9

Gaucher Disease and Its Treatment Options

Lunawati L. Bennett, Devipriya Mohan

Annals of Pharmacotherapy

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