Congenital thrombotic thrombocytopenic purpura

Synonyms: Congenital ADAMTS-13 deficiency | Congenital TTP | Familial TTP | Upshaw-Schulman syndrome

A hereditary form of thrombotic thrombocytopenic purpura (TTP) characterized by profound peripheral thrombocytopenia microangiopathic hemolytic anemia (MAHA) and single or multiple organ failure of variable severity.

Data from Orphanet are used to provide information on a disease's name, synonym(s), and overview. Reference: Access aggregated data from Orphanet at Orphadata. Orphadata: Free access data from Orphanet. © INSERM 1999. Available on http://www.orphadata.org. Data version April 2026

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Clinical Trials

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Congenital thrombotic thrombocytopenic purpura

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Clinical Trials

Newly diagnosed with
Congenital thrombotic thrombocytopenic purpura?