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Updated: Mar 14, 2026

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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针对阿波脂蛋白 (a) 的反感性寡核酸:两个随机,双盲,安慰剂对照,剂量范围的试验

Nicholas J Viney1, Julian C van Capelleveen2, Richard S Geary1

  • 1Ionis Pharmaceuticals, Carlsbad, CA, USA.

Lancet (London, England)
|September 26, 2016
PubMed
概括

在临床试验中,IONIS-APO (a) Rx和IONIS-APO (a) -LRx两种新型反感性寡核酸可显著降低高脂蛋白 (a) (Lp (a)). 这些疗法有望缓解与高Lp (a) 相关的心血管疾病风险.

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