DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Glanzmann thrombasthenia 1 — screening already-approved drugs against its 4-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleGlanzmann thrombasthenia 1 maps to a 4-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 glanzmann thrombasthenia 1 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
coagulation factor VII (F7) — F7 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 2rdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5PAG · 1.36 Å · ligand (2R)-2-hydroxy-N-[[3-[5-hydroxy-4-(1H-pyrrolo[3,2-c]pyridin-2-yl)pyrazol-1-yl]phenyl]methyl]-3-methylbutanamide (7YJ). Experimental structure, not a prediction.
What the evidence adds up to
Glanzmann thrombasthenia is a rare inherited autosomal recessive platelet functional disorder characterised by mucocutaneous haemorrhage of varying severity due to qualitative defects of platelets. Diagnosis is difficult because it closely mimics other bleeding disorders and can be confirmed only after investigations and exclusion of others. Laboratory analysis in a group of 7 patients showed decreased or absent platelet aggregation (less than 10%) with all physiologic agonists (ADP, collagen, epinephrin, arachidonic acid) together with a normal agglutination response to ristocetin. In three of those patients diagnosis was confirmed by flow cytometry.
Allogeneic haematopoietic stem-cell transplantation is described as the only currently curative procedure, but carries major risks, particularly in adults. In one reported adult patient who developed antiplatelet antibodies and became refractory to any pharmacological treatment, the patient died after transplantation. No other curative treatment is described in these reports.
Supportive care with platelet transfusion and proper counselling is the standard treatment. With careful supportive care, the prognosis is described as very good. One case report describes a 14-year-old HBsAg-positive adolescent male with Glanzmann thrombasthenia, and another describes a 13-year-old female. Bleeding history in the group of 7 patients included both mucosal and postsurgery bleeds.
What is still missing is any controlled trial data for any drug therapy, any evidence that platelet transfusion is effective in patients who have developed antiplatelet antibodies, and any stratification of patients by antibody status or bleeding severity to guide treatment decisions. No drug repurposing candidate is mentioned in any of these abstracts.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Journal of Indian Society of Pedodontics and Preventive Dentistry · 2014 · 15 citations · open access
Hematological and surgical management in Glanzmann′s thrombasthenia: A case report
AbstractGlanzmann's thrombasthenia (GT) is a rare, congenital, and moderate to severe platelet disorder. The bleeding time is increased, due to lack of platelet aggregation, since the patients with GT have deficient or dysfunctional integrin membrane glycoproteins IIb and IIIa essential for platelet aggregation. Children with GT are mostly diagnosed very early in life due to the spontaneous and unexplained mucocutaneous bleeding. It is quite a challenging task when any surgery is indicated for children with GT. This case report is about the medical and surgical management of an 11-year-old girl diagnosed with Glannzmann's thrombasthenia who had to undergo a maxillary cyst enucleation.
Clinical Case Reports · 2017 · 14 citations · open access
Allogeneic hematopoietic cell transplantation in an adult patient with Glanzmann thrombasthenia
AbstractGlanzmann thrombasthenia is a rare bleeding disorder that can present life-threatening bleeding. Our patients develop antiplatelet antibodies that become refractory to any pharmacological treatment. Allogeneic hematopoietic stem-cell transplantation is the only currently curative procedure, but has major risks mainly in adult; indeed, our patient died.
Journal of Indian Society of Pedodontics and Preventive Dentistry · 2007 · 4 citations · open access
Glanzmann′s thrombasthenia associated with HBsAg-positive child: A case report
AbstractGlanzmann's thrombasthenia is a rare hemorrhagic disorder characterized by prolonged bleeding time and diminished clot retraction. The disease is marked by frequent mucocutaneous hemorrhage which is mainly due to qualitative defects of platelets. A case of a 14-year-old HBsAg-positive adolescent male with Glanzmann's thrombasthenia has been presented.
Faridpur Medical College Journal · 2021 · 1 citations · open access
Glanzmann's Thrombasthenia: A rare platelet functional disorder
AbstractGlanzmann's Thrombasthenia (GT) is a rare inherited autosomal recessive platelet functional disorder. Due to the deficiency of platelet function, it manifests as a bleeding disorder characterized by mucocutaneous hemorrhage of varying severity. It is difficult to diagnose as it closely mimics with others bleeding disorder, so it can be diagnosed after investigations & exclusion of others. Treatment is supportive care with platelet transfusion & proper counseling. With careful supportive care, GT has a very good prognosis. In this report, we describe a 13 years old female with Glanzmann Thrombasthenia. Faridpur Med. Coll. J. 2020;15(2): 103-105
VIMS Health Science Journal · 2021 · 1 citations · open access
A Rare Case of Bleeding Disorder: Glanzmann’s Thrombasthenia
AbstractGlanzmann’s thrombasthenia is an extremely rare autosomal recessive inherited bleeding disorder characterized by defective platelet aggregation leading to prolonged bleeding time. Patients may present with easy bruising, purpura, epistaxis, menorrhagia and gingival bleeding. Though the disease is rare, the prognosis is usually excellent with supportive care. Here, we report the case of Glanzmann’s thrombasthenia in a young female who presented with complaints of epistaxis and a history of easy bruising. The patient improved with symptomatic and supportive care. The patient got discharged and is doing well under regular follow-up.
Hematology Transfusion and Cell Therapy · 2021 · 0 citations · open access
THE CLINICAL PICTURE AND LABORATORY WORK-UP OF GLANZMANN THROMBASTHENIA
AbstractCase report We present the clinical picture and laboratory work-up of Glanzmann thrombasthenia, based on a group of 7 patients. Bleeding history was significant in all patients and included both mucosal and postsurgery bleeds. Laboratory analysis revealed decreased or absent platelet aggregation (< 10%) with all physiologic agonists (ADP, collagen, epinephrin, arachidonic acid) together with normal agglutination response to ristocetin. In three patients diagnosis was confirmed by flow cytometry.
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.
