A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design
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- 作者：K.V. Truxala ; d ; i ; H. Fua ; i ; D.M. McCartya ; i ; K.A. McNallyc ; e ; i ; K.L. Kunklera ; N.A. Zumbergef ; j ; L. Martinf ; j ; S.C. Aylwardg ; k ; L.N. Alfanoa ; K.M. Berrya ; L.P. Lowesa ; M. Corridoreh ; l ; C. McKeeh ; K.L. McBrideb ; d ; i ; K.M. Flanigana ; g ; i ; k ; kevin.flanigan@nationwidechildrens.org" class="auth_mail" title="E-mail the corresponding author
- 关键词：AAV9 ; adeno-associated virus ; serotype 9 ; ABAS-II ; Adaptive Behavior Assessment System ; Second Edition ; ALT ; alanine aminotransferase ; aPTT ; activated partial thromboplastin time ; AST ; aspartate aminotransferase ; BBB ; blood-brain barrier ; CBC ; complete blood count ; CBCL ; Achenbach Child Behavior Checklist ; CTCAE ; Common Terminology Criteria for Adverse Events ; GAG ; glycosaminoglycan ; GGT ; gamma-glutamyl transferase ; INR ; international normalized ratio ; IRB ; institutional review board ; MPS III ; m
- 刊名：Molecular Genetics and Metabolism
- 出版年：2016
- 出版时间：November 2016
- 年：2016
- 卷：119
- 期：3
- 页码：239-248
- 全文大小：874 K
文摘
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Robust outcome measures are needed for clinical trials in the mucopolysaccharidoses types 3A and 3B
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Leiter nonverbal IQ standard scores significantly decline over 6 month intervals in patients less than 7 years old
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Vineland-II composite scores decline significantly over 6 month intervals.
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Cognitive and behavioral testing can distinguish declines in patients with MPS over periods relevant to clinical trials