Dystrophin restoration by exon 53 skipping in patients with Duchenne muscular dystrophy after viltolarsen treatment: phase 2 study update

Clemens P.; Rao V.; Connolly A.; Zaidman C.; Harper A.; Mah J.; Morgenroth L.; Yamashita T.; Hoffman E.

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Dystrophin restoration by exon 53 skipping in patients with Duchenne muscular dystrophy after viltolarsen treatment: phase 2 study update

机译：EXON 53在viltolarsen治疗后患有Duchenne肌营养不良患者的EXON 53患者的营养不良症恢复：第2期研究更新

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《Neuromuscular disorders: NMD》 |2019年第1期|共2页
作者
Clemens P.; Rao V.; Connolly A.; Zaidman C.; Harper A.; Mah J.; Morgenroth L.; Yamashita T.; Hoffman E.;
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作者单位

Univ Pittsburgh Pittsburgh PA USA;

Lurie Childrens Hosp Chicago IL USA;

Washington Univ St Louis MO 63110 USA;

Washington Univ St Louis MO 63110 USA;

Virginia Commonwealth Univ Richmond VA 23284 USA;

Alberta Childrens Prov Gen Hosp Calgary AB Canada;

TRiNDS Kensington PA USA;

NS Pharma Paramus NJ USA;

SUNY Binghamton Binghamton NY USA;

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原文格式 PDF
正文语种 eng
中图分类神经病学;
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1. Dystrophin restoration by exon 53 skipping in patients with Duchenne muscular dystrophy after viltolarsen treatment: phase 2 study update [J] . Clemens P., Rao V., Connolly A., Neuromuscular disorders: NMD . 2019,第Suppla1期

机译：EXON 53在viltolarsen治疗后患有Duchenne肌营养不良患者的EXON 53患者的营养不良症恢复：第2期研究更新
2. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. [J] . Cirak S, Arechavala Gomeza V, Guglieri M, The Lancet . 2011,第9791期

机译：系统性二重氨基磷酸吗啉代低聚物治疗后的Duchenne肌营养不良患者的外显子跳跃和肌营养不良蛋白的恢复：一项开放性，2期，剂量递增研究。
3. Exon 53 skipping of the dystrophin gene in patients with Duchenne muscular dystrophy by systemic administration of NS-065/NCNP-01: A phase 1, dose escalation, first-in-human study [J] . Komalki H., Nagata T., Saito T., Neuromuscular disorders: NMD . 2015,第Suppla2期

机译：通过全身给药NS-065 / NCNP-01，杜氏肌营养不良患者的肌营养不良蛋白基因的外显子53跳过：第1阶段，剂量递增，首次在人体中进行研究
4. Targeted MS~2 Quantitation of Exon Skipping Restored Dystrophin in a Mouse Model of Duchenne Muscular Dystrophy [C] . Kristy J. Brown, Kitipong Uaesoontrachoon, Aiping Zhang, ASMS Conference on Mass Spectrometry and Allied Topics . 2014

机译：杜南肌营养不良小鼠模型中外显子跳水恢复营养蛋白的靶向MS〜2
5. Experimental and Computational Studies of Structural Differences between Alternative Exon Skipped Repairs for Duchenne Muscular Dystrophy [D] . Ma, Manyuan 2018

机译：实验和计算研究杜兴氏肌营养不良的其他外显子跳过修复之间的结构差异。
6. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label phase 2 dose-escalation study [O] . Sebahattin Cirak, Virginia Arechavala-Gomeza, Michela Guglieri, -1

机译：系统性二氨基磷酸二氨基吗啉代低聚物治疗后的杜兴氏肌营养不良患者的外显子跳跃和肌营养不良蛋白的恢复：一项开放性2期剂量递增研究
7. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study [O] . Cirak, S, Arechavala-Gomeza, V, Guglieri, M, 2011

机译：系统性二氨基氨基磷酸吗啉代低聚物治疗后的杜兴氏肌营养不良患者的外显子跳跃和肌营养不良蛋白的恢复：一项开放性，2期，剂量递增研究

Dystrophin restoration by exon 53 skipping in patients with Duchenne muscular dystrophy after viltolarsen treatment: phase 2 study update

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