In a paper published on Feb. 21, 2024, in Science Translational Medicine, researchers from the Versiti Blood Research Institute described how they successfully controlled bleeding for months in hemophilia A models using a siRNA therapy that targeted plasminogen.
In a recent study, researchers from Technische Universität Dresden and affiliated organizations evaluated the novel small-molecule inhibitor of the CBP/p300 HAT domain, A-485, as a potential therapeutic candidate for the treatment of hematological cancers and other pathologies related to blood leukocyte compartment.
Bleeding of unknown cause is a group of rare disorders that are still difficult to accurately diagnose. A case report on a patient with hematoma in the perineal region after her first delivery was presented.
The rare hereditary fibrinogen disorder hypodysfibrinogenemia is characterized by fibrinogen defects, which can cause thrombotic and hemorrhagic phenotypes that are not always predicted by routine coagulation tests. Researchers from Academic Hospital Maastricht aimed to characterize the genetic profile of a family with hypodysfibrinogenemia and predict bleeding and/or thrombotic phenotypes in asymptomatic family members using innovative testing.
Bleeding of unknown cause (BUC) is a diagnosis of exclusion, and it is common for these patients to have congenital platelet function disorders. Whole-exome sequencing may help reach a more accurate diagnosis in these cases.
E-Therapeutics plc has offered a pipeline update, following the nomination of novel target genes, which have yielded promising results in preclinical studies.
Dysregulation of the kallikrein-kinin system (KKS) is involved in hereditary angioedema (HA) pathophysiology; thus, the inhibition of factor XIIa (FXIIa), a central mediator of angioedema and initiator of KKS, is a therapeutic strategy to prevent and treat HA attacks.
A sustained antiplatelet effect plus target selectivity are the two major requirements for developing new antithrombotic therapies. Increasing the levels of cAMP in the platelets by the action of a prostacyclin receptor (PTGIR) agonist is a possible approach for this purpose. Researchers from the University of Michigan have presented preclinical data on their PTGIR agonist CS-585, which has shown higher blood stability, as a potential therapeutic for thrombosis.