Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6 months of therapy in cats using different IV infusion durations
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- 作者：Therese Ruanea ; Mark Haskinsa ; Alphonsus Chengd ; Ping Wanga ; Gustavo Aguirreb ; Van W. (Rafe) Knox IVc ; Yulan Qid ; Troy Tompkinsd ; Charles A. O'Neilld ; CO'Neill@bmrn.com" class="auth_mail" title="E-mail the corresponding author
- 关键词：Mucopolysaccharidosis VI ; Maroteaux&ndash ; Lamy syndrome ; Galsulfase ; Recombinant human N-acetylgalactosamine 4-sulfatase ; Enzyme replacement therapy ; Infusion duration
- 刊名：Molecular Genetics and Metabolism
- 出版年：2016
- 出版时间：February 2016
- 年：2016
- 卷：117
- 期：2
- 页码：157-163
- 全文大小：808 K
文摘
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rhASB is an approved enzyme replacement therapy for patients with MPS VI.
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Some clinicians wish to treat patients using reduced infusion times.
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Feline MPS VI model was used to compare the pharmacodynamics and ADME of 2- v 4- hr IV infusions of rhASB.
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No pharmacodynanmic differences were seen between 2- v 4-hour rhASB infusions given to MPS VI cats for 26 weeks.
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Clinical studies of shorter rhASB infusion times are appropriate for MPS VI patients without infusion-associated reactions.
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