ArticleTherapeutics and clinical risk management2008
Review of miglustat for clinical management in Gaucher disease type 1.
Article in Therapeutics and clinical risk management, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
37 citing papers in PubMed, 1 synthesis or guideline pooled it, 88 citations in OpenAlex.
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- Exploring Small-Molecule Inhibitors of Glucosidase II: Advances, Challenges, and Therapeutic Potential in Cancer and Viral Infection.International journal of molecular sciences · 2025Review
- Therapeutic Targeting of Viral N-Glycosylation Modification: From Molecular Mechanisms to Clinical Application Prospects.Infectious diseases and therapy · 2025Review
- QSP-Copilot: An AI-Augmented Platform for Accelerating Quantitative Systems Pharmacology Model Development.CPT: pharmacometrics & systems pharmacology · 2025Article
- Elovl1 inhibition reduced very long chain fatty acids in a mouse model of adrenoleukodystrophy.iScience · 2025Article
- Development of iminosugar-based glycosidase inhibitors as drug candidates for SARS-CoV-2 virus via molecular modelling and in vitro studies.Journal of enzyme inhibition and medicinal chemistry · 2024Article
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- A zebrafish model of combined saposin deficiency identifies acid sphingomyelinase as a potential therapeutic target.Disease models & mechanisms · 2023Article
- Glucosylceramide Synthase Inhibitors Induce Ceramide Accumulation and Sensitize H3K27 Mutant Diffuse Midline Glioma to Irradiation.International journal of molecular sciences · 2023Article
- Glucosylceramide is essential for Heartland and Dabie bandavirus glycoprotein-induced membrane fusion.PLoS pathogens · 2023Article
- Mysterious sphingolipids: metabolic interrelationships at the center of pathophysiology.Frontiers in physiology · 2023Review
- Sphingolipids in neurodegenerative diseases.Frontiers in neuroscience · 2023Review
- A novel mouse model of CMT1B identifies hyperglycosylation as a new pathogenetic mechanism.Human molecular genetics · 2022Article
- Glycosphingolipids in Diabetes, Oxidative Stress, and Cardiovascular Disease: Prevention in Experimental Animal Models.International journal of molecular sciences · 2022Review
- GM1 Gangliosidosis-A Mini-Review.Frontiers in genetics · 2021Review
- Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience.Molecular genetics & genomic medicine · 2020Article
- Cheminformatics Tools for Analyzing and Designing Optimized Small-Molecule Collections and Libraries.Cell chemical biology · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gaucher disease is a progressive lysosomal storage disorder caused by the deficiency of glucocerebrosidase, and characterized by intralysosomal storage of glucosylceramide that leads to dysfunction in multiple organ systems. Intravenous enzyme replacement with imiglucerase is the accepted standard for treatment of symptomatic patients and has been effective in reducing many of the signs and symptoms of type I Gaucher disease in the majority of patients without serious adverse effects. An alternative therapeutic approach is substrate reduction therapy with N-butyldeoxynojirimycin (NB-DNJ) (miglustat; Zavesca((R))), an imino sugar that reversibly inhibits glucosylceremide synthase and reduces intracellular storage of glucosylceramide. Miglustat was recently approved in Europe and the United States for symptomatic patients with mild to moderate clinical manifestations for whom enzyme replacement therapy is not an option. This review article discusses the results of clinical studies and use of miglustat as a therapeutic agent in patients with type I Gaucher disease.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.