DeCure for Nephrogenic syndrome of inappropriate antidiuresis
DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for nephrogenic syndrome of inappropriate antidiuresis — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleNephrogenic syndrome of inappropriate antidiuresis maps to a 1-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 nephrogenic syndrome of inappropriate antidiuresis 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
arginine vasopressin receptor 2 (AVPR2) — AVPR2 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 clrdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7DW9 · 2.6 Å · ligand CHOLESTEROL (CLR). Experimental structure, not a prediction.
What the evidence adds up to
A 2009 report on a family with nephrogenic syndrome of inappropriate antidiuresis (NSIAD) described 30-year follow-up on two patients, both hemizygous for the R137C mutation on the AVPR2 gene. The proband and a maternal uncle presented in infancy with severe recurrent hyponatremia and a clinical picture consistent with SIADH but without elevated ADH. The proband’s mother was an asymptomatic carrier. The report notes successful treatment with oral urea during infancy and self-regulated fluid restriction in adult life, and describes a favourable long-term outcome.
A 2020 paediatric case of AVPR2-related NSIAD reported a male neonate diagnosed by DNA sequencing. The authors state that oral urea produced a complete correction of hyponatremia. They suggest that sequencing of AVPR2 should be performed in newborns with prolonged euvolemic hyponatremia, hypo-osmolality, high urinary sodium, and normal or undetectable AVP levels, and that oral urea is a safe and effective treatment option in infants until they are grown-up.
A 2021 review of two paediatric cases of NSIAD from activating AVPR2 mutations reports that the first two cases of oral furosemide treatment were described in that article. The authors state that furosemide helped maintain normal serum sodium after fluid restriction and sodium supplementation had shown limited effect. The review notes that NSIAD presents with hyponatremia without other specific findings, and that genetic testing aids early diagnosis and timely treatment.
What remains missing is prospective data comparing urea, furosemide, and fluid restriction head-to-head in children and adults, long-term safety data for furosemide in this specific condition, and any controlled trial that stratifies patients by mutation type or age at diagnosis. No drug has been tested in a randomised fashion for NSIAD.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
International Journal of Pediatric Endocrinology · 2009 · 18 citations · open access
Long-Term Outcomes in a Family with Nephrogenic Syndrome of Inappropriate Antidiuresis
AbstractWe report a familial case of the nephrogenic syndrome of inappropriate antidiuresis (NSIAD), including 30-year followup data on two patients. The proband and one maternal uncle presented in their infancy with severe recurrent hyponatremia, and clinical pictures consistent with the syndrome of inappropriate antidiuretic hormone (SIADH) in the absence of an elevated ADH level. They were both confirmed to be hemizygous for the R137C mutation on the V2R gene (AVPR2), the same locus of the gain of function mutation demonstrated in the original reports of this condition. The proband's mother was identified as an asymptomatic carrier of this X-linked condition. Our case describes a favourable long-term outcome for NSIAD, in particular, successful treatment with oral urea during the infancy period, and with self-regulated precautions on fluid intake into adult life.
[Diagnosis and follow-up of 2 cases of pediatric nephrogenic syndrome of inappropriate antidiuresis resulting from activating mutation in AVPR2 and literature review].
AbstractNSIAD presented with hyponatremia without any other specific presentations. Genetic testing for variants in AVPR2 is helpful for early diagnosis and timely treatment. The first two cases of oral furosemide treatment were reported by the article which helped to maintain a normal serum sodium level after limiting fluid intake and supplementing sodium which showed limited effect.
Journal of the Korean Society of Pediatric Nephrology · 2020 · 0 citations · open access
A Pediatric Case of AVPR2-related Nephrogenic Syndrome of Inappropriate Antidiuresis
AbstractNephrogenic syndrome of inappropriate antidiuresis (NSIAD) is a rare X-linked genetic condition caused by a gain-of-function mutation of arginine vasopressin receptor 2 gene, AVPR2. We report the case of a male neonate diagnosed with NSIAD based on his DNA sequence of the AVPR2 gene and the clinical course. He demonstrated a complete correction of hyponatremia using oral urea. We suggest that (1) sequencing analysis of the AVPR2 gene ought to be done in newborns with prolonged euvolemic hyponatremia, hypo-osmolality, high urinary sodium and normal/low or undetectable AVP levels, and that (2) oral urea is a safe and effective treatment option in infants diagnosed with NSIAD until the patients are grown-up.
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.
