The multinational, open-label, controlled, randomized, crossover, phase 3 TAK-755 (NCT03393975) trial demonstrated superior efficacy and safety of recombinant ADAMTS13 as prophylaxis in patients with ...
Including biomarkers for non-ADAMTS13 in routine clinical testing could benefit diagnosis and follow-up in patients with immune-mediated thrombotic thrombocytopenic purpura (TTP). Patients with immune ...
Congenital thrombotic thrombocytopenic purpura (TTP) results from severe hereditary deficiency of ADAMTS13. The efficacy and safety of recombinant ADAMTS13 and standard therapy (plasma-derived ...
Adzynma is a purified recombinant form of the ADAMTS13 protein designed to replace the missing or deficient ADAMTS13 enzyme. The Food and Drug Administration (FDA) has approved Adzynma for ...
The FDA said it has received postmarketing reports of neutralizing antibodies to ADAMTS13, including one death, in patients with congenital thrombotic thrombocytopenic purpura (cTTP) who were treated ...
cTTP is an Ultra-rare Blood Clotting Disorder Associated with Life-Threatening Acute Events and Debilitating Chronic Symptoms Takeda Continues 70-plus Year Legacy of Driving Innovation for the Rare ...
ADAMTS13 is a circulating zinc metalloprotease that cleaves the hemostatic glycoprotein von Willebrand factor (VWF) in a shear-dependent manner. Deficiency in ADAMTS13, owing to genetic mutations or ...
Hepatic platelet accumulation in PA-ILI was confirmed in both patients and mice. Deficiency of ADAMTS13 plays a critical role in platelet accumulation in PA-ILI, suggesting that ADAMTS13 could be a ...
Pyrrolizidine alkaloids (PAs), widely distributed in plants, are known to induce liver failure. Hepatic platelet accumulation has been reported during the progression of PA-induced liver injury ...
Supported by a grant (1R01HL166246, to Dr. Bendapudi) from the National Institutes of Health and by the Luick Family Fund of the Department of Pathology at Massachusetts General Hospital. Disclosure ...
“People living with cTTP face serious, life-threatening health challenges, and until today, were without any approved treatment specifically indicated for their disease,” said Julie Kim, president, ...