Adam Feuerstein ✡️· @adamfeuerstein · X·· 4 小时前AI 评分42
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在杜氏肌营养不良症中，一种有前景的疗法只有少数幸运儿能用上 @Jasonmmast 从可能接受这些疗法的孩子们——以及那些无法接受（至少短期内无法接受）的孩子们——的视角，审视来自 $NVS 和 $DYN 的下一代外显子跳跃疗法。 https://www.statnews.com/2026/10/07/muscular-dystrophy-exon-skipping-drugs-novartis-dyne/
正文
In Duchenne muscular dystrophy, a promising therapy is available to a fortunate few
@Jasonmmast examines the next-generation of exon-skippers from $NVS and $DYN through the eyes of kids who may receive them, and those that won't (at least not for a long time.)
https://www.statnews.com/2026/10/07/muscular-dystrophy-exon-skipping-drugs-novartis-dyne/
来源：Adam Feuerstein ✡️ · x.com