A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design

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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. Truxal^a ; ^d ; ⁱ ; H. Fu^a ; ⁱ ; D.M. McCarty^a ; ⁱ ; K.A. McNally^c ; ^e ; ⁱ ; K.L. Kunkler^a ; N.A. Zumberge^f ; ^j ; L. Martin^f ; ^j ; S.C. Aylward^g ; ^k ; L.N. Alfano^a ; K.M. Berry^a ; L.P. Lowes^a ; M. Corridore^h ; ^l ; C. McKee^h ; K.L. McBride^b ; ^d ; ⁱ ; K.M. Flanigan^a ; ^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

•: Robust outcome measures are needed for clinical trials in the mucopolysaccharidoses types 3A and 3B
•: Leiter nonverbal IQ standard scores significantly decline over 6 month intervals in patients less than 7 years old
•: Vineland-II composite scores decline significantly over 6 month intervals.
•: Cognitive and behavioral testing can distinguish declines in patients with MPS over periods relevant to clinical trials

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