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Updated: Jun 18, 2026

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
05:16

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

Published on: May 7, 2020

筋縮に対する介入:臨床に入ってくる分子薬

Kate Bushby1, Hanns Lochmüller, Stephen Lynn

  • 1Institute of Human Genetics, Newcastle University, Newcastle upon Tyne, UK. kate.bushby@ncl.ac.uk

Lancet (London, England)
|December 1, 2009
PubMed
まとめ

筋肉ジストロフィーは,慢性的な障害を引き起こす珍しい遺伝疾患です. 決定的な治療法はまだ未定ですが,診断と管理の進歩により,罹患者のケアが改善されています.

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