HIGHLIGHTS
- who: Junjie Sun from the Keele University, United Kingdom have published the research work: History of development of the life-saving drug ``Nusinersen`` in spinal muscular atrophy, in the Journal: (JOURNAL)
- what: This approach has not yet been successful in clinical trials. This study provided a clear research target for this lethal disease and marked the formal entry of SMA treatment into the gene period. This research provided initial evidence that MOE-modified ASO 10-27 could effectively correct the splicing of SMN2 in animal tissues, although it required intracerebroventricular (ICV) or intrathecal (IT) injection . . .
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