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

Drug Repurposing and Lysosomal Storage Disorders: A Trick to Treat.

Genes - 25 Feb 2024

Hay Mele Bruno, Rossetti Federica, Cubellis Maria Vittoria, Monticelli Maria, Andreotti Giuseppina

Abstract excerpt

Rare diseases, or orphan diseases, are defined as diseases affecting a small number of people compared to the general population. Among these, we find lysosomal storage disorders (LSDs), a cluster of rare metabolic diseases characterized by enzyme mutations causing abnormal glycolipid storage. Drug repositioning involves repurposing existing approved drugs for new therapeutic applications, offering advantages in...

Read the complete abstract on PubMed

Topics

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

Share to Drug Repositioning
Share to Lysosomal Storage Diseases
Share to Lysosomes

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Mutation

Source

Open in PubMed Open full text

Identifiers

PMID: 38540351
DOI: 10.3390/genes15030290
PMCID: PMC10970111

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Drug Repurposing and Lysosomal Storage Disorders: A Trick to Treat.PMID 38540351Read ↗

FDA orphan drug designations for lysosomal storage disorders – a cross sectional analysisSemantic proximity Read ↗

Clinical pharmacology considerations for first‐in‐human clinical trials for enzyme replacement therapySemantic proximity Read ↗

Drug Repurposing in Rare Diseases: An Integrative Study of Drug Screening and Transcriptomic Analysis in Nephropathic CystinosisSemantic proximity Read ↗

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

Ten plus one challenges in diseases of the lysosomal system.Semantic proximity Read ↗

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

Evolving therapies in neuronopathic LSDs: opportunities and challenges.Semantic proximity Read ↗

Lysosomal storage disorders: old diseases, present and future challenges.Semantic proximity Read ↗

The Lysosome: A Potential Therapeutic Juncture between the COVID-19 Pandemic and Niemann-Pick Type C DiseaseSemantic proximity Read ↗

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

The discovery of medicines for rare diseases.Semantic proximity Read ↗

Hutchinson-Gilford progeria syndrome: Rejuvenating old drugs to fight accelerated ageingSemantic proximity Read ↗

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