DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for asphyxiating thoracic dystrophy 5 — 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 moduleAsphyxiating thoracic dystrophy 5 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 asphyxiating thoracic dystrophy 5 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
WD repeat domain 19 (WDR19) — WDR19 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 apo structuredrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8BBG · 3.5 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
A 13-month-old patient with Jeune's asphyxiating thoracic dystrophy was treated with a methyl-methacrylate prosthesis but died of respiratory distress two months after surgery. Two siblings with the condition were found to have hypoplastic lungs and abnormal phospholipid composition in airway secretions, with one showing abnormally low crying vital capacity and the other a markedly reduced peak flow rate. These findings suggest the respiratory impairment is not solely due to restricted chest expansion but also involves intrinsic lung hypoplasia and biochemical abnormalities.
Several surgical approaches have been reported. A 25-year-old male underwent a bilateral correction that eliminated chest wall depression and increased thoracic volume, described as a reasonable choice for that patient type. For a 3-year-old with type II disease (non-cylindrical chest with serious depressions), median thoracic expansion combined with a Nuss procedure achieved good results. However, for type I patients (cylindrical chest with slight depressions), median thoracic expansion alone was noted to be not ideal due to persistent depression effects.
A 4-year-old girl who underwent corrective surgery for thoracic deformity experienced severe respiratory distress and multiple complications. The authors of that report recommend that the optimal age for corrective surgery is between 6 and 12 years, and that for children under 6 the decision should be carefully evaluated. No drug therapy appears in any of these abstracts.
What is still missing is any pharmacological treatment for the underlying lung hypoplasia or phospholipid abnormality, prospective surgical trials with standardised outcome measures, and a clear understanding of which patients benefit from surgery at which age. The evidence consists entirely of case reports and small series, with no controlled comparisons.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
European Journal of Pediatric Surgery · 1998 · 28 citations
Jeune's Asphyxiating Thoracic Dystrophy of the Newborn
AbstractA 13-month-old patient with Jeune's thoracic asphyxiating dystrophy, was surgically treated using a methyl-methacrylate (acrylic) prosthesis. Although postoperative recovery was fast, the patient died of respiratory distress two months following surgery. Limitations of currently available surgical techniques and the need for long-term results will be discussed.
Journal of Surgical Case Reports · 2022 · 9 citations · open access
Bilateral correction of asphyxiating thoracic dystrophy
AbstractSeveral operations for asphyxiating thoracic dystrophy (ATD) have been used previously, but they all have disadvantages. We report a 25-year-old male ATD patient who had significant depressions on both sides of thorax. We designed a special operation for him, which had not only eliminated the depression, but also increased the volume of the thorax. The results show that this operation is a reasonable choice for this kinds of patient.
International Journal of Surgery Science · 2022 · 6 citations · open access
Surgical treatment of asphyxiating thoracic dystrophy with median thoracic expansion and Nuss procedure
AbstractAsphyxiating thoracic dystropy can be divided into two types: type I is cylindrical with slight depressions on chest wall, and type II is non-cylindrical with serious depressions on the chest wall. The nature of the depressions determines the choice of operation. For type I patients, the relatively reasonable operation is median thoracic expansion. However, due to the existence of depressions, the effect is not ideal. In order to eliminate the effect of depression, we designed a operation with an additional Nuss procedure to median thoracic expansion. We applied this operation to a 3-year-old patient and achieved good results.
Journal of Paediatrics and Child Health · 1987 · 6 citations
Hypoplastic lungs and abnormal phospholipids in asphyxiating thoracic dystrophy
AbstractThe respiratory impairment of asphyxiating thoracic dystrophy previously has been attributed to slower growth of the ribs which reduces chest size and limits chest expansion during breathing. Two siblings with this condition are described. One was found to have an abnormally low crying vital capacity; in the other the peak flow rate was reduced markedly. Chest X-rays and ventilation-perfusion nuclear scans were suggestive of hypoplastic lungs. Nasopharyngeal aspirates of airway secretions were found to contain significantly less total phospholipids and differences in phospholipid composition in comparison with a normal control group. These findings raise the possibility that the lungs are hypoplastic and have an abnormal phospholipid content in asphyxiating thoracic dystrophy.
Interdisciplinary CardioVascular and Thoracic Surgery · 2024 · 0 citations · open access
Surgical timing for asphyxiating thoracic dystrophy
AbstractThis report describes a 4-year-old girl diagnosed with asphyxiating thoracic dystrophy who experienced severe respiratory distress and multiple complications after undergoing a corrective operation for a thoracic deformity. The optimal age for children with asphyxiating thoracic dystrophy to receive a corrective operation is between 6 and 12 years old. For children under 6 years old, the decision to undergo an operation should be carefully evaluated.
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.
