ReviewNature reviews. Drug discovery2013
Animal models for metabolic, neuromuscular and ophthalmological rare diseases.
Review in Nature reviews. Drug discovery, 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
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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.
- Widefield OCT angiography.Progress in retinal and eye research · 2025Review
- Implementation status of pharmacological studies in the development of orphan drugs.Orphanet journal of rare diseases · 2024Article
- Isolation and Proteomic Analysis of Mouse Serum Small Extracellular Vesicles for Individual Subject Analysis.Methods in molecular biology (Clifton, N.J.) · 2022Article
- The rapidly evolving view of lysosomal storage diseases.EMBO molecular medicine · 2021Review
- Regulatory Standards in Orphan Medicinal Product Designation in the EU.Frontiers in medicine · 2021Review
- Genes Regulating Spermatogenesis and Sperm Function Associated With Rare Disorders.Frontiers in cell and developmental biology · 2021Review
- Molecular Diagnosis and Novel Therapies for Neuromuscular Diseases.Journal of personalized medicine · 2020Article
- A putative silencer variant in a spontaneous canine model of retinitis pigmentosa.PLoS genetics · 2020Article
- The Value of Mouse Models of Rare Diseases: A Spanish Experience.Frontiers in genetics · 2020Article
- Advances in the Treatment of Neuronal Ceroid Lipofuscinosis.Expert opinion on orphan drugs · 2019Article
- Maternal Inheritance of a Recessive RBP4 Defect in Canine Congenital Eye Disease.Cell reports · 2018Article
- The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impact.European journal of human genetics : EJHG · 2017Article
- Drug discovery and development for rare genetic disorders.American journal of medical genetics. Part A · 2017Review
- Catheterization of the gallbladder: A novel mouse model of severe acute cholangitis.World journal of gastroenterology · 2017Article
- Integrative Systems Biology Investigation of Fabry Disease.Diseases (Basel, Switzerland) · 2016Article
- Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.Science translational medicine · 2016Article
- Disease models for the development of therapies for lysosomal storage diseases.Annals of the New York Academy of Sciences · 2016Review
- Model organisms in the fight against muscular dystrophy: lessons from drosophila and Zebrafish.Molecules (Basel, Switzerland) · 2015Review
- Establishing medical plausibility in the context of orphan medicines designation in the European Union.Orphanet journal of rare diseases · 2014Article
- Drosophila as a starting point for developing therapeutics for the rare disease Duchenne Muscular Dystrophy.Rare diseases (Austin, Tex.) · 2013Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Animal models are important tools in the discovery and development of treatments for rare diseases, particularly given the small populations of patients in which to evaluate therapeutic candidates. Here, we provide a compilation of mammalian animal models for metabolic, neuromuscular and ophthalmological orphan-designated conditions based on information gathered by the European Medicines Agency's Committee for Orphan Medicinal Products (COMP) since its establishment in 2000, as well as from a review of the literature. We discuss the predictive value of the models and their advantages and limitations with the aim of highlighting those that are appropriate for the preclinical evaluation of novel therapies, thereby facilitating further drug development for rare diseases.
Indexed as
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23493083What 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.