Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases.

Journal of lipid research - 1 Jul 2014

Shayman James A, Larsen Scott D

Abstract excerpt

Glycosphingolipid (GSL) storage diseases have been the focus of efforts to develop small molecule therapeutics from design, experimental proof of concept studies, and clinical trials. Two primary alternative strategies that have been pursued include pharmacological chaperones and GSL synthase inhibitors. There are theoretical advantages and disadvantages to each of these approaches. Pharmacological chaperones are...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Glycoside Hydrolases
Share to Glycosphingolipids
Share to Lysosomal Storage Diseases

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Molecular Chaperones

Share to Mutation

Source

Open in PubMed Open full text

Identifiers

PMID: 24534703
DOI: 10.1194/jlr.R047167
PMCID: PMC4076080

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases.PMID 24534703Read ↗

Therapeutic strategies to ameliorate lysosomal storage disorders--a focus on Gaucher disease.Semantic proximity Read ↗

Carbohydrate-Processing Enzymes of the Lysosome: Diseases Caused by Misfolded Mutants and Sugar Mimetics as Correcting Pharmacological Chaperones.Semantic proximity Read ↗

Progress and potential of non-inhibitory small molecule chaperones for the treatment of Gaucher disease and its implications for Parkinson disease.Semantic proximity Read ↗

Update on treatment of lysosomal storage diseases.Semantic proximity Read ↗

Treatment of lysosomal storage disorders: successes and challengesSemantic proximity Read ↗

New strategies for the treatment of lysosomal storage diseases (Review)Semantic proximity Read ↗

Lysosomal storage diseases: current therapies and future alternativesSemantic proximity Read ↗

Therapeutic Approaches in Lysosomal Storage DiseasesSemantic proximity Read ↗

Novel beta-glucocerebrosidase chaperone compounds identified from cell-based screening reduce pathologically accumulated glucosylsphingosine in iPS-derived neuronal cells.Semantic proximity Read ↗

A counterintuitive approach to treat enzyme deficiencies: use of enzyme inhibitors for restoring mutant enzyme activity.Semantic proximity Read ↗

[Chemical chaperone therapy for lysosomal storage diseases].Semantic proximity Read ↗

Candidate molecules for chemical chaperone therapy of GM1-gangliosidosis.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.