3 Topic Commentaries

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

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  Peter Marks, M.D., Ph.D.

  Biologics regulation

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  U.S. Food and Drug Administration

  The FDA remains deeply committed in our efforts to help facilitate the development and approval of safe and effective therapies for patients with rare diseases. Without treatment, cTTP is ultimately fatal. Today’s approval reflects important progress in the development of much-needed treatment options for patients affected by this life-threatening disorder.

  Source
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  David Ginsburg, M.D.

  Genetics (molecular genetics of blood clotting)

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  University of Michigan Life Sciences Institute and Medical School

  The big advantage of the recombinant protein, over human plasma, is that you’re just getting this pure, clean protein. With this new treatment, we know exactly what is going into the patient, and at exactly what dose, and we don’t risk exposing the patient to anything else that may be in human plasma.

  Source
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  Karen Vanhoorelbeke, Not specified in source

  Biochemistry

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  University of Leuven (KU Leuven), Belgium

  When these long VWF multimers are not cleaved by ADAMTS13, VWF-platelet aggregation occurs, leading to TTP.

  Source