Clinically meaningful improvements after gene therapy for aromatic L-amino acid decarboxylase deficiency (AADCd) in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) and correlation with Bayley-III scores and motor milestones.
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| Title: | Clinically meaningful improvements after gene therapy for aromatic L-amino acid decarboxylase deficiency (AADCd) in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) and correlation with Bayley-III scores and motor milestones. |
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| Authors: | Hwu, Wuh-Liang1 (AUTHOR), Lee, Hui-Min1 (AUTHOR), Peipert, John Devin2 (AUTHOR), Zhang, Rongrong3 (AUTHOR), Werner, Christian4 (AUTHOR), Sierra, J. Rafael5 (AUTHOR), O'Connell, Thomas6 (AUTHOR), Woolley, Jonathan J.6 (AUTHOR), Crowell, Marjorie6 (AUTHOR), Wang, Antonia5 (AUTHOR), Tomazos, Ioannis5 (AUTHOR) ytomazos@ptcbio.com |
| Source: | Orphanet Journal of Rare Diseases. 2/7/2025, Vol. 20 Issue 1, p1-9. 9p. |
| Database: | Academic Search Ultimate |
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