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Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura

by Xiao, Shan , López-Fernández, M. Fernanda , Cataland, Spero R. , Hassenpflug, Wolf-Achim , Zhang, Pinghai , Scully, Marie , Coppo, Paul , Dossier, Claire , Bhattacharya, Indranil , Cronin, Michael , Matsumoto, Masanori , Kremer Hovinga, Johanna A. , Mellgård, Björn , Patwari, Parth , Patel, Munjal , Li, Hong , Ortel, Thomas L. , Biebuyck, Nathalie , Wang, Linda T. , Kentouche, Karim , Knöbl, Paul , Antun, Ana , Windyga, Jerzy

in ADAMTS13 Protein - administration & dosage / ADAMTS13 Protein - adverse effects / ADAMTS13 Protein - deficiency / ADAMTS13 Protein - genetics / Adolescent / Adult / Adverse events / Antibodies / Body weight / Child / Child, Preschool / Childhood Diseases / Coagulation / Congenital diseases / Cross-Over Studies / Disease prevention / Female / Hematology / Humans / Hypothesis testing / Male / Middle Aged / Oncology / Patients / Pediatrics / Pharmacokinetics / Plasma / Purpura / Purpura, Thrombotic Thrombocytopenic - congenital / Purpura, Thrombotic Thrombocytopenic - drug therapy / Purpura, Thrombotic Thrombocytopenic - genetics / Recombinant Proteins - administration & dosage / Recombinant Proteins - adverse effects / Thrombocytopenia / Thrombocytopenic purpura / Thrombotic thrombocytopenic purpura / Young Adult

2024

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Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura

2024

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2024

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Journal Article

Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura

Xiao, Shan,

López-Fernández, M. Fernanda,

Cataland, Spero R.,

Hassenpflug, Wolf-Achim,

Zhang, Pinghai,

Scully, Marie,

Coppo, Paul,

Dossier, Claire,

Bhattacharya, Indranil,

Cronin, Michael,

Matsumoto, Masanori,

Kremer Hovinga, Johanna A.,

Mellgård, Björn,

Patwari, Parth,

Patel, Munjal,

Li, Hong,

Ortel, Thomas L.,

Biebuyck, Nathalie,

Wang, Linda T.,

Kentouche, Karim,

Knöbl, Paul,

Antun, Ana,

Windyga, Jerzy

2024

Overview

Congenital thrombotic thrombocytopenic purpura results from hereditary deficiency of ADAMTS13. Prophylactic administration of recombinant ADAMTS13 achieved normal blood levels with limited toxicity and no neutralizing antibodies.

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Publisher

Massachusetts Medical Society

Subject

ADAMTS13 Protein - administration & dosage

/ ADAMTS13 Protein - adverse effects

/ ADAMTS13 Protein - deficiency

/ ADAMTS13 Protein - genetics

/ Adolescent

/ Adult

/ Adverse events