Prenatal treatment of spinal muscular atrophy.
作者信息
Servais Laurent
机构信息
MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK; Neuromuscular Reference Center, Department of Pediatrics, University Hospital Liège & University of Liège, Liège, Belgium.
出版信息
Mol Ther. 2025 May 7;33(5):1862-1863. doi: 10.1016/j.ymthe.2025.04.010. Epub 2025 Apr 18.
Abstract
摘要
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本文引用的文献
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Newborn screening programs for spinal muscular atrophy worldwide in 2023.
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Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy.
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A reassessment of spinal cord pathology in severe infantile spinal muscular atrophy: Reassessment of spinal cord pathology.
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Treatment Options in Spinal Muscular Atrophy: A Pragmatic Approach for Clinicians.
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Neurofilament light protein as a biomarker for spinal muscular atrophy: a review and reference ranges.
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Longitudinal developmental profile of newborns and toddlers treated for spinal muscular atrophy.
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