ArticleAmerican journal of medical genetics. Part C, Seminars in medical genetics2025
CURE ID: A Platform to Collect Real-World Treatment Data for Drug Repurposing in Rare Genetic Disorders.
Tahsin Farid, Maura R Z Ruzhnikov, Mili Duggal, Keyla C Tumas, Shira Strongin, Eric Sid, Sarah R Fuchs, Leonard Sacks, Dominique C Pichard, Catherine Pilgrim-Grayson and 2 more
Abstract read
In one paragraphArticle in American journal of medical genetics. Part C, Seminars in medical genetics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
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.
2 · The registryThe 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.
3 · Its place in the literatureWho cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
4 · The recordCorrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
5 · Who and what moneyAuthors and funding
12 authors.
Tahsin FaridDivision of Preclinical Innovation, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Rockville, Maryland, USA.ORCID https://orcid.org/0000-0002-3787-6079 Maura R Z RuzhnikovDivision of Rare Diseases and Medical Genetics, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.ORCID https://orcid.org/0000-0001-9610-1612 Keyla C TumasDivision of Preclinical Innovation, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Rockville, Maryland, USA.ORCID https://orcid.org/0009-0000-7448-5395 Shira StronginDivision of Preclinical Innovation, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Rockville, Maryland, USA.ORCID https://orcid.org/0000-0002-4819-6343 Eric SidDivision of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.ORCID https://orcid.org/0000-0001-7697-3026 Sarah R FuchsDivision of Rare Diseases and Medical Genetics, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.ORCID https://orcid.org/0009-0008-1583-7299 Dominique C PichardDivision of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.ORCID https://orcid.org/0000-0002-6492-622X Catherine Pilgrim-GraysonDivision of Rare Diseases and Medical Genetics, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.ORCID https://orcid.org/0000-0002-4264-8636 Funding
Informatics Research CoreZICTR000410 · NCATS · NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES · PI MATHÉ, EWY · 2021 to 2025
$11.7MNeglected Tropical Disease CURE DatabaseZIATR000056 · NCATS · NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES · PI MATHÉ, EWY · 2015 to 2025
$5.8MIntramural NIH HHS ZIA TR000056Intramural NIH HHS ZIC TR000410NIH HHS ZIA-TR000056NIH HHS ZIC-TR000410
6 · The paper itselfAbstract
Rare diseases collectively affect millions of Americans, but less than 5% have approved treatments, and new drug development remains limited. For such diseases, drug repurposing may be an effective strategy to find new treatment options. In the rare genetic disorder community, drugs are frequently prescribed off-label. This information is rarely available for research, but if captured, could be leveraged to accelerate the identification of candidate drugs to be evaluated for safety and efficacy of the treatment of rare diseases. CURE ID is a publicly available treatment registry that collects real-world treatment data directly from healthcare providers, patients, and care partners in a consistent format. By aggregating this information, CURE ID can generate hypotheses for follow-up targeted research of repurposed drugs, potentially leading to the approval of these drugs for new indications. The success of the platform is predicated on its adoption in the rare disease community and routinely reporting treatment experiences to CURE ID.
Indexed as
Drug RepositioningGenetic Diseases, InbornRare DiseasesHumansRegistriesCURE IDdrug repurposingrare diseasesrare genetic disordersreal‐world data
Identifiers
PMID41496707
PMCPMC12822485
What OpenQuestion holds
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