Recombinant ADAMTS13: An Enzyme Replacement Therapy for the Management of Congenital Thrombotic Thrombocytopenic Purpura - Top-Institutions - MDSpire

Recombinant ADAMTS13: An Enzyme Replacement Therapy for the Management of Congenital Thrombotic Thrombocytopenic Purpura

Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare autosomal recessive condition that causes deficiency of the von Willebrand factor (vWF)-cleaving metalloprotease, ADAMTS13. Recombinant ADAMTS13 (rADAMTS13) is the first FDA-approved product for management of cTTP. This article reviews the pharmacology, pharmacokinetics, efficacy, safety, dosing, administration, and implications for advanced practitioners of rADAMTS13.

  • By

  • Taylor P. Robichaux

  • Kathryn E. Dane

  • P. Christopher Parish

  • Justin R. Arnall

  • Donald C. Moore

  • January 9, 2026

  • 15 min

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Top Institutions in Urology

Brief introduction explaining scope and methodology.

  • #1

    University of Florida College of Medicine
    University of Florida College of Medicine

    Gainesville, Florida

    Key Differentiators

    • Hematology

  • #2

    University of Pennsylvania
    University of Pennsylvania

    Philadelphia, Pennsylvania

    Key Differentiators

    • Hematology

  • #3

    University of California, Los Angeles
    University of California, Los Angeles

    Los Angeles, California

    Key Differentiators

    • Hematology

  • #4

    City of Hope
    City of Hope

    Duarte, California

    Key Differentiators

    • Hematology

  • #5

    University College London
    University College London

    London, United Kingdom

    Key Differentiators

    • Hematology

Original Source(s)

Recombinant ADAMTS13: An Enzyme Replacement Therapy for the Management of Congenital Thrombotic Thrombocytopenic Purpura

  • by Taylor P. Robichaux, Kathryn E. Dane, P. Christopher Parish, Justin R. Arnall, Donald C. Moore

  • January 9, 2026

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