ArticleFuture medicinal chemistry2014
The discovery of medicines for rare diseases.
Article in Future medicinal chemistry, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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.
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Who cites it
14 citing papers in PubMed.
- Developing Treatments for Rare Diseases on a Shoestring.GEN biotechnology · 2023Article
- Small Molecule-Based Enzyme Inhibitors in the Treatment of Primary Hyperoxalurias.Journal of personalized medicine · 2021Review
- The Resurrection of Phenotypic Drug Discovery.ACS medicinal chemistry letters · 2020Article
- Review
- Structure-Based Identification and Characterization of Inhibitors of the Epilepsy-Associated KiScience · 2020Article
- Preclinical and Clinical Epigenetic-Based Reconsideration of Beckwith-Wiedemann Syndrome.Frontiers in genetics · 2020Review
- A Screen Using iPSC-Derived Hepatocytes Reveals NADCell reports · 2018Article
- Opportunities and challenges in phenotypic drug discovery: an industry perspective.Nature reviews. Drug discovery · 2017Review
- Industrializing rare disease therapy discovery and development.Nature biotechnology · 2017Article
- Incentives for Starting Small Companies Focused on Rare and Neglected Diseases.Pharmaceutical research · 2016Article
- A brief review of recent Charcot-Marie-Tooth research and priorities.F1000Research · 2015Review
- The value of translational biomarkers to phenotypic assays.Frontiers in pharmacology · 2014Article
- Collaboration for rare disease drug discovery research.F1000Research · 2014Article
- Collecting rare diseases.F1000Research · 2014Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
Abstract
There is a pressing need for new medicines (new molecular entities; NMEs) for rare diseases as few of the 6800 rare diseases (according to the NIH) have approved treatments. Drug discovery strategies for the 102 orphan NMEs approved by the US FDA between 1999 and 2012 were analyzed to learn from past success: 46 NMEs were first in class; 51 were followers; and five were imaging agents. First-in-class medicines were discovered with phenotypic assays (15), target-based approaches (12) and biologic strategies (18). Identification of genetic causes in areas with more basic and translational research such as cancer and in-born errors in metabolism contributed to success regardless of discovery strategy. In conclusion, greater knowledge increases the chance of success and empirical solutions can be effective when knowledge is incomplete.
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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.