Dosing & Uses
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Congenital Thrombotic Thrombocytopenic Purpura
Pending FDA approval for enzyme replacement therapy of congenital thrombotic thrombocytopenic purpura (cTTP), an ADAMTS13 gene deficiency disorder
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Pharmacology
Mechanism of Action
Congenital thrombotic thrombocytopenic purpura (cTTP), is an ADAM metallopeptidase with thrombospondin type 1 motif 13 (ADAMTS13) gene deficiency disorder
ADAMTS13 enzyme splices von Willebrand factor to regulate its interaction with platelets; this action is involved in platelet adhesion to form temporary clots at the site of an injury
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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.