Miglustat

Last Updated: 04/28/2026

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Found 444 publications

A case of Joubert Syndrome and NPC1 mutation in a 7-year-old girl: presented with neuromotor developmental delay and ataxia.

A case of Joubert Syndrome and NPC1 mutation in a 7-year-old girl: presented with neuromotor developmental delay and ataxia.

Journal: Neurocase

Published: April 14, 2026

Correction: Miglustat as a Treatment for Adults with Tangier Disease Neuropathy: The MUSTANG N‑of‑1 Trial with 21 months Clinical Observation.

Correction: Miglustat as a Treatment for Adults with Tangier Disease Neuropathy: The MUSTANG N‑of‑1 Trial with 21 months Clinical Observation.

Journal: Neurology and therapy

Published: March 12, 2026

Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data.

Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data.

Journal: Journal of comparative effectiveness research

Published: February 27, 2026

Identification of serum protein biomarkers in individuals with Niemann-Pick disease, type C1.

Identification of serum protein biomarkers in individuals with Niemann-Pick disease, type C1.

Journal: medRxiv : the preprint server for health sciences

Published: February 26, 2026

Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.

Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.

Journal: Health technology assessment (Winchester, England)

Published: February 16, 2026

Adverse Event Profile Differences Between Eliglustat and Miglustat: A Pharmacovigilance Study using the U.S. Food and Drug Administration Adverse Event Reporting System.

Adverse Event Profile Differences Between Eliglustat and Miglustat: A Pharmacovigilance Study using the U.S. Food and Drug Administration Adverse Event Reporting System.

Journal: Drug research

Published: February 05, 2026

N-Alkyl Derivatives of Deoxynojirimycin (DNJ) as Antiviral Agents: Overview and Update.

N-Alkyl Derivatives of Deoxynojirimycin (DNJ) as Antiviral Agents: Overview and Update.

Journal: Molecules (Basel, Switzerland)

Published: December 15, 2025

Altered bone structure in Niemann-Pick Type C1 mice, especially in females.

Altered bone structure in Niemann-Pick Type C1 mice, especially in females.

Journal: Bone

Published: December 02, 2025

Comprehensive review of recent advances in Pompe disease: pathogenesis, management, and future directions.

Comprehensive review of recent advances in Pompe disease: pathogenesis, management, and future directions.

Journal: Frontiers in neurology

Published: November 29, 2025

First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease.

First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease.

Journal: Journal of neurology

Published: November 19, 2025

Investigation of dipyridamole-elicited signaling in the brain of Niemann Pick type C mice: A multi-omic study.

Investigation of dipyridamole-elicited signaling in the brain of Niemann Pick type C mice: A multi-omic study.

Journal: Brain research bulletin

Published: October 30, 2025

Drug-induced urinary incontinence in pediatric patients: A disproportionality analysis of the FDA Adverse Event Reporting System.

Drug-induced urinary incontinence in pediatric patients: A disproportionality analysis of the FDA Adverse Event Reporting System.

Journal: Medicine

Published: October 28, 2025

Showing 1-12 of 444

Last Updated: 04/28/2026