Recombinant ADAMTS-13: first-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura.

M, Scully; P, Knöbl; K, Kentouche; L, Rice; J, Windyga; R, Schneppenheim; Hovinga JA, Kremer; M, Kajiwara; Y, Fujimura; C, Maggiore; J, Doralt; C, Hibbard; L, Martell; B, Ewenstein

doi:10.1182/blood-2017-06-788026

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Recombinant ADAMTS-13: first-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura.

Shared by NobleBlocks on Sep 14, 2017 • 12:00 AM UTC

Authors:

Scully M

Knöbl P

Kentouche K

Abstract

Safety, tolerability, and pharmacokinetics of recombinant ADAMTS-13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13; BAX 930; SHP655) were investigated in 15 patients diagnosed with severe congenital ADAMTS-13 deficiency (plasma ADAMTS-13 activity max [U/mL]) and a...

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