A 20-year Clinical and Genetic Neuromuscular Cohort Analysis in Lebanon: An International Effort [0.03%]
黎巴嫩20年神经肌肉队列的临床和遗传分析：一项国际努力
Andre Megarbane,Sami Bizzari,Asha Deepthi et al.
Andre Megarbane et al.
Background: Clinical and molecular data on the occurrence and frequency of inherited neuromuscular disorders (NMD) in the Lebanese population is scarce. O...
The Effect of Immunomodulatory Treatments on Anti-Dystrophin Immune Response After AAV Gene Therapy in Dystrophin Deficient mdx Mice [0.03%]
AAV基因治疗后免疫调节治疗对杜氏肌营养不良mdx小鼠抗 dystrophin 免疫反应的影响
Ning Li,Joanna E Parkes,Rita Spathis et al.
Ning Li et al.
Background: AAV-based gene therapy is an attractive approach to treat Duchenne muscular dystrophy (DMD) patients. Although the long-term consequences of a gene therapy approach for DMD are unknown, there is evidence in bo...
Effects of Chronic, Maximal Phosphorodiamidate Morpholino Oligomer (PMO) Dosing on Muscle Function and Dystrophin Restoration in a Mouse Model of Duchenne Muscular Dystrophy [0.03%]
慢性最大剂量膦酰吗啉寡核苷酸(PMO)对杜氏肌营养不良小鼠模型肌肉功能和 dystrophin 恢复的影响
Margaret E Benny Klimek,Maria Candida Vila,Katie Edwards et al.
Margaret E Benny Klimek et al.
Background: Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is currently used in clinical development to treat Duchenne muscular dystrophy (DMD), with four exon-skipping drugs achieving regulatory appr...
Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy [0.03%]
外显子跳跃治疗后对 dystrophin 和 dystroglycan 复合蛋白周转的质询
James S Novak,Rita Spathis,Utkarsh J Dang et al.
James S Novak et al.
Recently, the Food and Drug Administration granted accelerated approvals for four exon skipping therapies -Eteplirsen, Golodirsen, Viltolarsen, and Casimersen -for Duchenne Muscular Dystrophy (DMD). However, these treatments have only demon...
Rebuttal to: Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy, Verhaart et al. 2020 [0.03%]
对Verhaart等人2020年的《辛伐他汀治疗不能改善杜氏肌营养不良小鼠模型的肌肉病理生理》一文的反驳
N P Whitehead,M J Kim,K L Bible et al.
N P Whitehead et al.
Author's Response to: Rebuttal to: Simvastatin Treatment Does Not Ameliorate Muscle Pathophysiology in a Mouse Model for Duchenne Muscular Dystrophy, Verhaart et al. 2020 [0.03%]
作者回应：Verhaart等人2020年文章《辛伐他汀治疗不能改善杜氏肌营养不良小鼠模型的肌肉病理生理》的反驳意见
Annemieke Aartsma-Rus,Ingrid Verhaart,Dominic Wells
Annemieke Aartsma-Rus
Marine Theret,Marielle Saclier,Graziella Messina et al.
Marine Theret et al.
While skeletal muscle remodeling happens throughout life, diseases that result in its dysfunction are accountable for many deaths. Indeed, skeletal muscle is exceptionally capable to respond to stimuli modifying its homeostasis, such as in ...
Enhancing Interrogation of Skeletal Muscle Samples for Informative Quantitative Data [0.03%]
提高骨骼肌样本审讯的 informative quantitative data 的能力
Terence A Partridge
Terence A Partridge
Careful quantitative analysis of histological preparations of muscle samples is crucial to accurate investigation of myopathies in man and of interpretation of data from animals subjected to experimental or potentially therapeutic treatment...
Nertiyan Elangkovan,George Dickson
Nertiyan Elangkovan
Duchenne muscular dystrophy (DMD) is an X-linked, muscle wasting disease that affects 1 in 5000 males. Affected individuals become wheelchair bound by the age of twelve and eventually die in their third decade due to respiratory and cardiac...
Adult North Star Network (ANSN): Consensus Guideline For The Standard Of Care Of Adults With Duchenne Muscular Dystrophy [0.03%]
成人北极星网络（ANSN)：杜氏肌营养不良症患者的护理标准共识指导意见
R Quinlivan,B Messer,P Murphy et al.
R Quinlivan et al.
There are growing numbers of adults with Duchenne Muscular Dystrophy living well into their fourth decade. These patients have complex medical needs that to date have not been addressed in the International standards of care. We sought to c...