Rare & Orphan Lab · DeCure for X

DeCure for Hydranencephaly

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hydranencephaly — 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.

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The disease map

Disease moduleHydranencephaly 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 hydranencephaly 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

nudE neurodevelopment protein 1 (NDE1)NDE1 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 gtpdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 7E1T · 2.45 Å · ligand GUANOSINE-5'-TRIPHOSPHATE (GTP). Experimental structure, not a prediction.

What the evidence adds up to

A 2014 case report describes endoscopic choroid plexus coagulation (ECPC) as the preferred treatment for hydranencephaly but reports a previously unreported complication: arachnoid collapse. The authors state that the variable anatomy in hydranencephaly predisposes the procedure to this dangerous complication and discuss preventative measures. No survival data, response rates, or sample sizes are given; the report is based on a single case.

A 2020 review from Nepal states that hydranencephaly is a rare congenital disorder in which the cerebral cortex is absent and replaced by fluid, presenting as hydrocephalus and developmental delay. The review notes that overall prognosis is grim and that clinicians, especially in low- and middle-income countries, face a dilemma about whether to offer cerebrospinal fluid diversion. No quantitative outcomes are provided.

A separate 2020 case report describes anaesthetic considerations for an infant with hydranencephaly who underwent three procedures requiring anaesthesia between 5 months and 2 years of age. The authors note that medical advancements have prolonged life expectancy for these patients and that caring for them is becoming more routine. They emphasise the need for anticipation of difficult positioning, deliberate airway management, and attention to anaesthetic recovery. Again, no survival or response rates are given; the report covers a single patient.

What is still missing are prospective studies with defined outcome measures, any controlled trial of ECPC versus shunt placement, and systematic data on long-term survival and quality of life. Patient stratification by anatomy or comorbidities has not been attempted. Funding for multicentre registries or trials in this rare condition remains absent.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Turkish Neurosurgery · 2014 · 7 citations · open access

Hydranencephaly: considering prolonged survival and treatment by endoscopic choroid plexus coagulation

AbstractArachnoid collapse is a previously unreported complication of endoscopic choroid plexus coagulation (ECPC) treatment of hydranencephaly and is demonstrated in this case report. The variable anatomy found in hydranencephaly supports the use of ECPC as the preferred treatment option. However, the same anatomical anomalies predispose the procedure to this unique complication. A brief literature review of hydranencephaly has also been performed and is reported through the discussion. Neurosurgeons must be aware of this very dangerous complication and a discussion of preventative measures are made. The use of ECPC and the clinical expectations of treating patients with hydranencephaly is also discussed.

https://doi.org/10.5137/1019-5149.jtn.10453-14.1
Nepal Journal of Neuroscience · 2020 · 5 citations · open access

Hydranencephaly: Insights into Pathophysiology and Management

AbstractHydranencephaly is a rare and severe form of congenital disorder in which there is absence of cerebral cortex which is replaced by fluid. The presentation is in the form of hydrocephalus and developmental delay. There are various reports on possible etiopathogenesis and management. However, the overall prognosis is grim and clinicians especially in low and middle-income countries like Nepal often face a clinical judgement dilemma regarding management options to offer to the patient family. The ethical issue whether to offer cerebrospinal fluid diversion or not is always there. This review is aimed at discussing the various aspects of management of this pediatric neurosurgical problem.

https://doi.org/10.3126/njn.v17i1.28336
BMC Anesthesiology · 2020 · 0 citations · open access

Unveiling what is absent within: illustrating anesthetic considerations in a patient with hydranencephaly – a case report

AbstractBACKGROUND: Hydranencephaly is a rare and debilitating congenital condition in which most anesthesiologists are unfamiliar. Primary surgical treatment involves CSF diversion, though other palliative procedures requiring anesthesia are often required. With medical advancements and a resulting prolonged life expectancy, caring for these patients is becoming more routine. CASE PRESENTATION: We follow an infant with hydranencephaly over three different procedures requiring anesthesia from 5 months of age to 2 years, highlighting the various anesthetic considerations. CONCLUSIONS: Anticipation of difficult positioning, deliberate airway management, and attention to anesthetic recovery were all necessary to safely care for this patient. An understanding of the challenges this particular condition poses will help anesthesiologists provide the most safe and effective care when encountering these patients.

https://doi.org/10.1186/s12871-020-01142-3

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.