Functional assessment tools in children with Pompe disease: A pilot comparative study to identify suitable outcome measures for the standard of care
Author:
Publisher
Elsevier BV
Subject
Clinical Neurology,General Medicine,Pediatrics, Perinatology, and Child Health
Reference28 articles.
1. Infantile acid maltase deficiency;Griffin;Virchows Arch B Cell Pathol Incl Mol Pathol,1984
2. Pompe disease in infants and children;Kishnani;J Pediatr,2004
3. Pompe disease diagnosis and management guideline;Kishnani;Genet Med Off J Am Coll Med Genet,2006
4. Enzyme replacement therapy in classical infantile Pompe disease: results of a ten-month follow-up study;Klinge;Neuropediatrics,2005
5. Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations;Case;Am J Med Genet C Semin Med Genet,2012
Cited by 4 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献
1. Arginine:glycine amidinotransferase (AGAT) deficiency: an easy-to-miss treatable adult-onset myopathy;Practical Neurology;2024-02-13
2. Quantitative Motion Measurements Based on Markerless 3D Full-Body Tracking in Children with SMA Highly Correlate with Standardized Motor Assessments;Journal of Neuromuscular Diseases;2022-01-04
3. Spinal Muscular Atrophy: The Use of Functional Motor Scales in the Era of Disease-Modifying Treatment;Child Neurology Open;2021-01-01
4. New pharmacotherapies for genetic neuromuscular disorders: opportunities and challenges;Expert Review of Clinical Pharmacology;2019-07-02
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3