Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy

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  Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy

  By Hirofumi Komaki, Tetsuya Nagata, Takashi Saito, Satoru Masuda, Eri Takeshita, Masayuki Sasaki, Hisateru Tachimori, Harumasa Nakamura, Yoshitsugu Aoki, Shin’ichi Takeda

  Science Translational Medicine18 Apr 2018

  NS-065/NCNP-01, an antisense oligonucleotide that enables exon 53 skipping in the dystrophin gene, showed a favorable safety profile and promising pharmacokinetics in 10 patients with Duchenne muscular dystrophy.

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  Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy

  By Hirofumi Komaki, Tetsuya Nagata, Takashi Saito, Satoru Masuda, Eri Takeshita, Masayuki Sasaki, Hisateru Tachimori, Harumasa Nakamura, Yoshitsugu Aoki, Shin’ichi Takeda

  Science Translational Medicine18 Apr 2018

  NS-065/NCNP-01, an antisense oligonucleotide that enables exon 53 skipping in the dystrophin gene, showed a favorable safety profile and promising pharmacokinetics in 10 patients with Duchenne muscular dystrophy.

  • Supplementary Materials

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