Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
10833895 | Molecular Genetics and Metabolism | 2012 | 5 Pages |
Abstract
⺠Examined cognitive and motor skills in 17 infants with Pompe disease treated with ERT. ⺠Most subjects exhibited rapid gains in motor ability following the initiation of ERT. ⺠Cognitive scores of responders to ERT remained stable. ⺠Overall cognitive scores were within normal limits throughout the study.
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Authors
Gail A. Spiridigliozzi, James H. Heller, Laura E. Case, Harrison N. Jones, Priya S. Kishnani,