ReviewDrugs2002
New prospects for the treatment of lysosomal storage diseases.
Review in Drugs, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 100 citations in OpenAlex.
- Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing.Journal of the American Society of Nephrology : JASN · 2007Trial
- Applications of Recombinant DNA Technology in Medicine: A Comprehensive Review.Molecular biotechnology · 2026Review
- Pain Phenotypes in Rare Musculoskeletal and Neuromuscular Diseases.Neuroscience and biobehavioral reviews · 2021Review
- Article
- Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier.Proceedings of the National Academy of Sciences of the United States of America · 2013Article
- Chaperone therapy for neuronopathic lysosomal diseases: competitive inhibitors as chemical chaperones for enhancement of mutant enzyme activities.Perspectives in medicinal chemistry · 2009Article
- Article
- Efficient gene therapy-based method for the delivery of therapeutics to primate cortex.Proceedings of the National Academy of Sciences of the United States of America · 2009Article
- Genistin-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: An open-label, pilot study in 10 pediatric patients.Current therapeutic research, clinical and experimental · 2008Article
- Abnormalities in the hair morphology of patients with some but not all types of mucopolysaccharidoses.European journal of pediatrics · 2008Article
- Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome.Brain research · 2008Article
- Farber disease: clinical presentation, pathogenesis and a new approach to treatment.Pediatric rheumatology online journal · 2007Article
- Modest phenotypic improvements in ASA-deficient mice with only one UDP-galactose:ceramide-galactosyltransferase gene.Lipids in health and disease · 2006Article
- Direct multiplex assay of lysosomal enzymes in dried blood spots for newborn screening.Clinical chemistry · 2004Article
- Electrocardiographic and other cardiac anomalies in beta-glucuronidase-null mice corrected by nonablative neonatal marrow transplantation.Proceedings of the National Academy of Sciences of the United States of America · 2004Article
- Endocytic trafficking of glycosphingolipids in sphingolipid storage diseases.Philosophical transactions of the Royal Society of London. Series B, Biological sciences · 2003Review
- A historical perspective of the glycosphingolipids and sphingolipidoses.Philosophical transactions of the Royal Society of London. Series B, Biological sciences · 2003Article
- Modulators of ceramide metabolism sensitize colorectal cancer cells to chemotherapy: a novel treatment strategy.Journal of gastrointestinal surgery : official journal of the Society for Surgery of the Alimentary Tract · 2003Article
- Chemical chaperones increase the cellular activity of N370S beta -glucosidase: a therapeutic strategy for Gaucher disease.Proceedings of the National Academy of Sciences of the United States of America · 2002Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
Although individually rare, lysosomal storage disorders constitute a significant burden on society. To date, enzyme replacement therapy (ERT) has been the most successful therapeutic approach for lysosomal storage disorders. ERT reverses systemic manifestations of Gaucher disease but does not effectively treat the neurological complications. Recently, ERT produced a reduction of severe neuropathic pain, stabilisation of renal disease, and improved vascular function and structure in short-term, placebo-controlled trials in patients with Fabry's disease. Long-term studies are necessary to evaluate the full potential of ERT in this disease. In patients with Pompe disease, a fatal cardiac and skeletal muscle disorder, ERT improved cardiac function and structure, and increased overall muscle strength. It has already increased survival in a small number of affected infants. ERT also decreased liver and spleen size, joint mobility and quality of life in patients with mucopolysaccharidosis type I, but when the therapeutic protein is administered intravenously, it is unlikely to modify the neurological outcome in this or in other similar disorders. Bone marrow transplantation continues to be effective in Gaucher disease, in some forms of mucopolysaccharidosis and in mild forms of Krabbé disease, but it has high morbidity and mortality that limits its use in lysosomal storage disorders. Drugs that slow the rate of formation of accumulating glycolipids are being developed and one of them, OGT-918 (N-butyldeoxynojirimycin), is showing promise in patients with Gaucher disease. Gene therapy for lysosomal storage disorders holds promise as a replacement for the other therapies described here but requires much more development before clinical efficacy trials.
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What OpenQuestion holds
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.