ReviewPhilosophical transactions of the Royal Society of London. Series B, Biological sciences2003
Substrate reduction therapy in mouse models of the glycosphingolipidoses.
Review in Philosophical transactions of the Royal Society of London. Series B, Biological sciences, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04768166 (Phase 2 Pharmacological Trial to Evaluate the Safety of Miglustat Administration in Subjects With Spastic Paraplegia 11), which is not on this map. Cited by 24 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
The trial behind it
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.
Phase 2 Pharmacological Trial to Evaluate the Safety of Miglustat Administration in Subjects With Spastic Paraplegia 11 (TreatSPG11)
Who cites it
24 citing papers in PubMed.
- Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease.Journal of inherited metabolic disease · 2004Trial
- Induced Pluripotent Stem Cells for the Treatment of Lysosomal Storage Disorders.Journal of inherited metabolic disease · 2025Review
- Therapeutic developments for neurodegenerative GM1 gangliosidosis.Frontiers in neuroscience · 2024Review
- Gaucher disease - more than just a rare lipid storage disease.Journal of molecular medicine (Berlin, Germany) · 2022Review
- GM2 Gangliosidoses: Clinical Features, Pathophysiological Aspects, and Current Therapies.International journal of molecular sciences · 2020Review
- GBA1 mutations: Prospects for exosomal biomarkers in α-synuclein pathologies.Molecular genetics and metabolism · 2020Review
- New Approaches to Tay-Sachs Disease Therapy.Frontiers in physiology · 2018Review
- Article
- Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders.International journal of molecular sciences · 2016Review
- [Globosides as key players in the pathophysiology of Shiga toxin-associated acute kidney failure and Fabry disease].Der Pathologe · 2014Article
- Development of targeted therapies for Parkinson's disease and related synucleinopathies.Journal of lipid research · 2014Review
- Depletion of globosides and isoglobosides fully reverts the morphologic phenotype of Fabry disease.Cell and tissue research · 2014Article
- Therapeutic effects of stem cells and substrate reduction in juvenile Sandhoff mice.Neurochemical research · 2012Article
- Article
- Lysosomal storage of oligosaccharide and glycosphingolipid in imino sugar treated cells.Glycoconjugate journal · 2010Article
- Substrate deprivation therapy: a new hope for patients suffering from neuronopathic forms of inherited lysosomal storage diseases.Journal of applied genetics · 2007Review
- Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis.Proceedings of the National Academy of Sciences of the United States of America · 2003Article
- Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease.Philosophical transactions of the Royal Society of London. Series B, Biological sciences · 2003Review
- Small-molecule therapeutics for the treatment of glycolipid lysosomal storage disorders.Philosophical transactions of the Royal Society of London. Series B, Biological sciences · 2003Review
- Neurobiology and cellular pathogenesis of glycolipid storage diseases.Philosophical transactions of the Royal Society of London. Series B, Biological sciences · 2003Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Substrate reduction therapy uses small molecules to slow the rate of glycolipid biosynthesis. One of these drugs, N-butyldeoxynojirimycin (NB-DNJ), shows efficacy in mouse models of Tay-Sachs, Sandhoff and Fabry diseases. This offers the prospect that NB-DNJ may be of therapeutic benefit, at least in the juvenile and adult onset variants of these disorders. The infantile onset variants will require an additional enzyme-augmenting modality if the pathology is to be significantly improved. A second drug, N-butyldeoxyglactonojirimycin, looks very promising for treating storage diseases with neurological involvement as high systemic dosing is achievable without any side-effects.
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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.