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Updated: Jan 11, 2026

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

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针对杜申肌肉发育不良的儿科药物开发:欧盟监管视角

Sylvie Benchetrit1, Céline Chu1, Dimitrios Athanasiou2

  • 1Department of Europe and Innovation, Agence nationale de sécurité du médicament et des produits de santé (ANSM), Saint-Denis, France.

Journal of neuromuscular diseases
|November 18, 2025
PubMed
概括

这项研究回顾了欧洲药物管理局 (EMA) 对杜申肌肉缩症 (DMD) 的儿科调查计划 (PIP),为未来的药物开发提供了见解,并突出了这种罕见疾病的监管策略.

关键词:
杜切内 (Duchenne) 是一个非常有价值的品牌.杜申尼肌肉发育不良症 杜申尼肌肉发育不良症欧洲药品管理局 欧洲药品管理局药物开发是药物的发展.孤儿疾病是一种孤儿疾病.

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