ReviewTransactions of the American Clinical and Climatological Association2013
The design and clinical development of inhibitors of glycosphingolipid synthesis: will invention be the mother of necessity?
Review in Transactions of the American Clinical and Climatological Association, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 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
20 citing papers in PubMed, 33 citations in OpenAlex.
- Article
- Article
- Gaucher disease - more than just a rare lipid storage disease.Journal of molecular medicine (Berlin, Germany) · 2022Review
- Anti-inflammatory role of GM1 and other gangliosides on microglia.Journal of neuroinflammation · 2022Article
- Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease.Journal of inherited metabolic disease · 2020Article
- CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges.Frontiers in molecular biosciences · 2020Review
- Sphingolipid Mediators of Myocardial Pathology.Journal of lipid and atherosclerosis · 2020Review
- Emerging targeted strategies for the treatment of autosomal dominant polycystic kidney disease.Clinical kidney journal · 2018Review
- Pharmacotherapy of Gaucher Disease: Current and Future Options.P & T : a peer-reviewed journal for formulary management · 2018Article
- Why Are Diabetes Medications So Expensive and What Can Be Done to Control Their Cost?Current diabetes reports · 2017Review
- Protection against Shiga Toxins.Toxins · 2017Review
- Sphingolipids and phospholipids in insulin resistance and related metabolic disorders.Nature reviews. Endocrinology · 2017Review
- Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders.International journal of molecular sciences · 2016Review
- An increase in glucosylceramide synthase induces Bcl-xL-mediated cell survival in vinorelbine-resistant lung adenocarcinoma cells.Oncotarget · 2015Article
- Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease.Drug design, development and therapy · 2015Review
- The consequences of genetic and pharmacologic reduction in sphingolipid synthesis.Journal of inherited metabolic disease · 2015Review
- Eliglustat: first global approval.Drugs · 2014Review
- Evolving concepts in cancer therapy through targeting sphingolipid metabolism.Biochimica et biophysica acta · 2014Review
- The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases.Journal of lipid research · 2014Review
- The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher's Disease Type 1: A Systematic Review.Journal of research in pharmacy practiceReview
Corrections and comments
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Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
The treatment of glycosphingolipid storage diseases by synthesis inhibition was first proposed 40 years ago as an alternative approach to enzyme replacement therapy. We have pursued this strategy through the rational design of potent and selective inhibitors of glucosylceramide synthase, the first step in glycosphingolipid synthesis. Eliglustat tartrate was the result of these efforts and is currently the focus of phase 3 trials for type 1 Gaucher disease. Phase 2 studies showed a reduction in splenomegaly and hepatomegaly and improvements of anemia and thrombocytopenia at levels equivalent to or exceeding the historic response to imiglucerase. Structural analogues of eliglustat have also been designed that lack pgp-1 recognition and cross the blood brain barrier. These may have utility for central nervous system- based sphingolipidoses. Because glycosphingolipids are important regulators of receptor tyrosine kinases, glucosylceramide synthase inhibitors may also be beneficial for disorders such as type 2 diabetes mellitus and polycystic kidney disease.
Indexed as
Identifiers
23874009PMC3715929W19618507What 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.