ArticleFrontiers in medicine2024
Drug repurposing for rare: progress and opportunities for the rare disease community.
Article in Frontiers in medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 1 of them a synthesis that pooled it.
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
31 citing papers in PubMed, 1 synthesis or guideline pooled it, 45 citations in OpenAlex.
- A bibliometric analysis and systematic review of drug repurposing against drug-resistant ESKAPE pathogens: a particular focus onFrontiers in microbiology · 2025Pooled it
- Using Deep Learning Models of Gene Regulation to Guide Drug Prioritization.Pharmaceuticals (Basel, Switzerland) · 2026Article
- Using Deep Learning Models of Gene Regulation to Guide Drug Prioritization.bioRxiv : the preprint server for biology · 2026Article
- High-Throughput Screening of FDA-Approved Drugs for Antibacterial and Antibiofilm Activities Against Multidrug-resistantACS omega · 2026Article
- Article
- New pharmacotherapies for the erythropoietic protoporphyrias: an analysis of trial protocols from a patient perspective.Orphanet journal of rare diseases · 2025Article
- ZebraMap: A Multimodal Rare Disease Knowledge Map with Automated Data Aggregation & LLM-Enriched Information Extraction Pipeline.Diagnostics (Basel, Switzerland) · 2025Article
- How social pharmaceutical innovations are addressing problems of availability, accessibility and affordability of drugs for rare diseases.Orphanet journal of rare diseases · 2025Article
- Data-driven strategies for drug repurposing: insights, recommendations, and case studies.Briefings in bioinformatics · 2025Article
- A Genomic Sequencing Approach to Newborn Mass Screening and Its Opportunities.JAMA network open · 2025Article
- Advances in mass spectrometry of lipids for the investigation of Niemann-pick type C disease.Lipids in health and disease · 2025Review
- Computational Drug Repositioning in Cardiorenal Disease: Opportunities, Challenges, and Approaches.Proteomics · 2025Article
- Allosteric regulation of UBIAD1 trafficking from ER to Golgi revealed by chemical genetic screening.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Rare Disease Drug Repurposing.JAMA network open · 2025Article
- The chemical chaperone 4-phenylbutyric acid rescues molecular cell defects of COL3A1 mutations that cause vascular Ehlers Danlos Syndrome.Cell death discovery · 2025Article
- Using chanarin-dorfman syndrome patient fibroblasts to explore disease mechanisms and new treatment avenues.Orphanet journal of rare diseases · 2025Article
- Combined in silico/in vitro approaches for identifying modulators of the activity of the p.Tyr110Cys Carnitine O-Acetyltransferase (CRAT) variant associated to an early onset case of Leigh syndrome.Acta pharmacologica Sinica · 2025Article
- Debates over orphan drug pricing: a meta-narrative literature review.Orphanet journal of rare diseases · 2025Review
- Targeting Glucosylceramide Synthase: Innovative Drug Repurposing Strategies for Lysosomal Diseases.International journal of molecular sciences · 2025Article
- Rifampicin Repurposing Reveals Anti-Melanogenic Activity in B16F10 Melanoma Cells.Molecules (Basel, Switzerland) · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 8 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Repurposing is one of the key opportunities to address the unmet rare diseases therapeutic need. Based on cases of drug repurposing in small population conditions, and previous work in drug repurposing, we analyzed the most important lessons learned, such as the sharing of clinical observations, reaching out to regulatory scientific advice at an early stage, and public-private collaboration. In addition, current upcoming trends in the field of drug repurposing in rare diseases were analyzed, including the role these trends could play in the rare diseases' ecosystem. Specifically, we cover the opportunities of innovation platforms, the use of real-world data, the use of artificial intelligence, regulatory initiatives in repurposing, and patient engagement throughout the repurposing project. The outcomes from these emerging activities will help progress the field of drug repurposing for the benefit of patients, public health and medicines development.
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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.