New treatment in the pipeline for rare clotting disorder

A recombinant ADAMTS-13 is showing promise for the treatment of patients with congenital thrombotic thrombocytopenic purpura (cTTP). A phase 1 multi-centre study of the new therapy in patients with severe congenital ADAMTS-13 deficiency found it behaved similarly to the endogenous enzyme and restored ADAMTS-13 activity. The researchers observed normalisation of the structure of von Willebrand ...

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