A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged 2 to 5 years with nonsense mutation Duchenne muscular dystrophy

Riebling P.; Kong R.; OMara E.; Luo X.; Trifillis P.; Ong T.

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A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged 2 to 5 years with nonsense mutation Duchenne muscular dystrophy

机译：2至5年患者用非阵突变肌营养不良患者患者的患者安全和药代动力学的第2阶段试验

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《Neuromuscular disorders: NMD》 |2017年第2期|共1页
作者
Riebling P.; Kong R.; OMara E.; Luo X.; Trifillis P.; Ong T.;
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作者单位

PTC Therapeut Inc South Plainfield NJ USA;

PTC Therapeut South Plainfield NJ USA;

PTC Therapeut Inc South Plainfield NJ USA;

PTC Therapeut Inc South Plainfield NJ USA;

PTC Therapeut Inc South Plainfield NJ USA;

PTC Therapeut Inc South Plainfield NJ USA;

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正文语种 eng
中图分类神经病学;
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专利

1. A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged = 2 to 5 years with nonsense mutation Duchenne muscular dystrophy [J] . Kong R., OMara E., Luo X., Neuromuscular disorders: NMD . 2018,第Suppla1期

机译：Ataluren在患者患者中ataluren的安全和药代动力学的第二阶段试验。= 2至＆用非阵容突变5年杜鹃肌肉营养不良
2. A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged 2 to 5 years with nonsense mutation Duchenne muscular dystrophy [J] . Riebling P., Kong R., OMara E., Neuromuscular disorders: NMD . 2017,第Suppla2期

机译：2至5年患者用非阵突变肌营养不良患者患者的患者安全和药代动力学的第2阶段试验
3. A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged 2 to 5 years with nonsense mutation Duchenne muscular dystrophy [J] . Riebling P., Kong R., OMara E., Neuromuscular disorders: NMD . 2017,第Suppla2期

机译：2至5年患者用非阵突变肌营养不良患者患者的患者安全和药代动力学的第2阶段试验
4. Protocol of Determination of Fatigue Index to Muscular Training in Patients with Duchenne's Muscular Dystrophy [C] . F.I. Correa, J.C.F. Correa, R.C.F. Moura, International Society of Electrophysiology and Kinesiology . 2004

机译：Duchenne肌营养不良患者患者疲劳指数疲劳指数的疗法协议
5. Young Patient Education on Duchenne Muscular Dystrophy [D] . McFarland, Margaret Jane Atieno. 2020

机译：在Duchenne肌营养不良的年轻患者教育
6. Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy [O] . Richard S. Finkel, Kevin M. Flanigan, Brenda Wong, -1

机译：2a期研究无意义突变的杜兴氏肌营养不良症患者中ataluren介导的肌营养不良蛋白的产生
7. Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy. [O] . Richard S Finkel, Kevin M Flanigan, Brenda Wong, 2013

机译：2a期研究ataluren介导的肌营养不良蛋白生成患者无义突变Duchenne肌营养不良症。

A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged 2 to 5 years with nonsense mutation Duchenne muscular dystrophy

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