DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for congenital stationary night blindness — screening already-approved drugs against its 24-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCongenital stationary night blindness maps to a 24-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.
Research record
01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Current lead
No approved-drug candidate for congenital stationary night blindness is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
Molecular view
retinol binding protein 4 (RBP4) — RBP4 is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
Loading structure…
helix sheet {4-[2-(trifluoromethyldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6QBA · 1.8 Å · ligand 2-[({4-[2-(trifluoromethyl)phenyl]piperidin-1-yl}carbonyl)amino]benzoic acid (2T1). Experimental structure, not a prediction.
What the evidence adds up to
Seven of eight patients in a 1988 case series of X-linked congenital stationary night blindness presented with decreased acuity and nystagmus and did not complain of night blindness; the diagnosis was made only by electroretinogram and dark adaptation testing. Two of those patients showed pupillary constriction to darkness, a sign of retinal disease in young patients. The authors concluded that careful electrodiagnostic testing is needed for accurate genetic counselling.
Two later review articles, from 2011 and 2016, both state that patients who complain of seeing badly in the dark require a full ophthalmological workup, including a history to distinguish acquired from congenital night blindness, visual acuity, biomicroscopy, fundus examination, visual field testing, and possibly flash ERG. Both note that not all patients who complain of seeing badly in the dark are actually night blind, and that a full clinical workup can identify several causes of both congenital and acquired night blindness.
No treatment, drug, or intervention for congenital stationary night blindness is mentioned in any of these abstracts. No data on survival, response rates, or sample sizes beyond the seven patients in the 1988 series are provided. The 1988 paper does not report any attempt to treat or modify the condition.
What is missing is any clinical trial testing a drug for congenital stationary night blindness, any funding for such a trial, and any method of patient stratification that might distinguish the subset of patients who do not complain of nyctalopia from those who do. The abstracts offer no molecular target, no biomarker, and no rationale for repurposing any existing drug.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Archives of ophthalmology · 1946 · 205 citations
CONGENITAL MALFORMATIONS INDUCED IN RATS BY MATERNAL VITAMIN A DEFICIENCY
AbstractALTHOUGH congenital blindness of offspring of poorly nourished animals has often been described, early reports were specific neither in analysis of nutritional deficiency nor in description of ocular defects. Most of observations were made on cattle, and Moore, Huffman and Duncan 1 have reviewed literature pertaining to this field. Two types of congenital blindness can be distinguished: the true vitamin A type, which is obviously a severe form of xerophthalmia, 2 and another type which is associated with constriction of optic foramen. 3 The latter, characterized by a dilated pupil and absence of inflammation of external structures of eye, is due to atrophy of optic nerve caused by its passage through optic foramen, apparently because of bony pressure. 1 It was difficult to attribute this congenital ocular defect to maternal vitamin A deficiency until Wolbach and Bessey
A CLINICAL METHOD FOR DETERMINING MODERATE DEGREES OF VITAMIN A DEFICIENCY
AbstractThe accepted and easily recognized clinical evidences of vitamin A deprivation are associated only with marked degrees of vitamin A deficiency. Night blindness is one of these several clinical phenomena, but it is one that is not recognized by an ordinary routine examination and one that usually is considered only when the complaint is made by the patient. A routine test for night blindness probably would reveal many more cases than are now suspected. It might be expected also that a suitable method of examination would detect night blindness, which is present in such a mild degree that the afflicted individual is entirely unaware of its existence. Since the etiology of night blindness in these cases is presumably the same, regardless of its degree, it should be possible to detect even moderate deficiency of vitamin A by means of appropriate tests. Our purpose in this presentation is to show that
Congenital Stationary Night Blindness Presenting as Leber's Congenital Amaurosis
AbstractTwo siblings with autosomal-recessive congenital stationary night blindness were clinically blind in infancy. Both had markedly abnormal electroretinograms that, in the first child, led consultants at two university centers to make the diagnosis of Leber's congenital amaurosis. The patients had intermittent nystagmus and esotropia, but good photopic vision developed eventually. Scotopic vision was clearly defective in each child. Refractive error in both patients was close to emetropic in early infancy but became myopic by 1 year of age. Congenital stationary night blindness must be considered in the differential diagnosis of the blind infant.
X-Linked Congenital Stationary Night Blindness With Myopia and Nystagmus Without Clinical Complaints of Nyctalopia
AbstractSeven of eight patients presented initially or were followed for decreased acuity and nystagmus without complaints of night blindness. The diagnosis of congenital stationary night blindness was established with electroretinogram and dark adaptation testing. Careful electrodiagnostic testing is needed to provide accurate genetic counseling. Two patients showed pupillary constriction to darkness which is a sign of retinal disease in young patients.
AbstractSummary Purpose Patients with complaints of seeing badly in the dark need a full ophthalmological workup. Methods Acquired versus congenital nightblindness has to be questioned in a full anamnesis. Visual acuity, biomicroscopy and fundus examination are essential followed by visual field testing and possibly visual electrophysiology (flash ERG). Results Several causes of congenital and acquired nightblindness can be found. Not all patients complaining of seeing badly in the dark are ‘nightblind’. Conclusion A full clinical ophthalmological work up can identify adequately several causes of night blindness.
AbstractAbstract Purpose Patients with complaints of seeing badly in the dark need a full ophthalmological workup. Methods Acquired versus congenital nightblindness has to be questioned in a full anamnesis. Visual acuity, biomicroscopy and fundus examination are essential followed by visual field testing and possibly visual electrophysiology (flash ERG) Results Several causes of congenital and acquired nightblindness can be found. Not all patients complaining of seeing badly in the dark are "nightblind" Conclusion A full clinical ophthalmological work up can identify adequately several causes of night blindness
Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
