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Substance / Medication

Miglustat

SNOMED 412296005UMLS C1321596

Overview

Active Ingredient
miglustat
RxNorm CUI
402316
Labeler: Navinta LLCUpdated: 2025-12-31T00:00:00.000ZFull label on DailyMed

Contraindications

When this intervention should not be used

None.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Chronic non-neuropathic Gaucher's disease
Kerasin thesaurismosis
Gaucher's disease

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data.
Shohet Simon, Hummel Noemi, Fu Shuai et al. · J Comp Eff Res · 2024
PMID: 39287071Meta-AnalysisFull text (PMC)
Efficacy and safety of miglustat in the treatment of GM2 gangliosidosis: A systematic review.
Mansouri Vahid, Tavasoli Ali Reza, Khodarahmi Masoud et al. · Eur J Neurol · 2023
PMID: 37209042Meta-Analysis
Dysphagia as a risk factor for mortality in Niemann-Pick disease type C: systematic literature review and evidence from studies with miglustat.
Walterfang Mark, Chien Yin-Hsiu, Imrie Jackie et al. · Orphanet J Rare Dis · 2012
PMID: 23039766Meta-AnalysisFull text (PMC)
Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL.
Kushlaf Hani, Díaz-Manera Jordi, Bratkovic Drago et al. · Muscle Nerve · 2025
PMID: 40342075RCT
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.
Kishnani Priya S, Byrne Barry J, Claeys Kristl G et al. · J Patient Rep Outcomes · 2024
PMID: 39535661RCTFull text (PMC)
A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat.
Remenova Tatiana, Morand Olivier, Amato Dominick et al. · Orphanet J Rare Dis · 2015
PMID: 26084276RCTFull text (PMC)
Changes in the atherogenic profile of patients with type 1 Gaucher disease after miglustat therapy.
Puzo J, Alfonso P, Irun P et al. · Atherosclerosis · 2010
PMID: 19959168RCT
Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase.
van Giersbergen Paul L M, Dingemanse Jasper · J Clin Pharmacol · 2007
PMID: 17720777RCT
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study.
Patterson Marc C, Vecchio Darleen, Prady Helena et al. · Lancet Neurol · 2007
PMID: 17689147RCT
An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease.
Roberts Mark E, Proskorovsky Irina, Guyot Patricia et al. · Adv Ther · 2025
PMID: 40920287ObservationalFull text (PMC)

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Miglustat (substance)
SNOMED CT
412296005
UMLS CUI
C1321596
RxNorm CUI
402316
Labeler
Navinta LLC

Clinical Data

This intervention maps to 3 entities in the Ltrl knowledge graph.

3
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.