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

作者：Kristen V. Truxal, Haiyan Fu, Douglas M. McCarty, Kelly A. McNally, Krista L. Kunkler, Nicholas A. Zumberge, Lisa C. Martin, Shawn C. Aylward, Lindsay N. Alfano, Katherine Berry, Linda Pax Lowes, Marco Corridore, Christopher T. McKee, Kim L. McBride, Kevin M. Flanigan · 发表于：Molecular Genetics and Metabolism · 年份：2016 · DOI：10.1016/j.ymgme.2016.08.002 · 被引用次数：51 · 研究领域：Lysosomal Storage Disorders Research、Retinopathy of Prematurity Studies、Glycogen Storage Diseases and Myoclonus