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Early treatment is a lifeline for infants with SMA
Nature Medicine ( IF 58.7 ) Pub Date : 2022-07-15 , DOI: 10.1038/s41591-022-01889-x Charlotte J Sumner 1, 2 , Thomas O Crawford 1, 3
Nature Medicine ( IF 58.7 ) Pub Date : 2022-07-15 , DOI: 10.1038/s41591-022-01889-x Charlotte J Sumner 1, 2 , Thomas O Crawford 1, 3
Affiliation
In the phase 3 SPR1NT trial, pre-symptomatic gene therapy demonstrated impressive clinical outcomes in infants with a genetic diagnosis of spinal muscular atrophy (SMA); long-term safety follow-up of these patients must now be a key priority.
中文翻译:
早期治疗是 SMA 婴儿的生命线
在 3 期 SPR1NT 试验中,症状前基因治疗在基因诊断为脊髓性肌萎缩症 (SMA) 的婴儿中表现出令人印象深刻的临床结果;对这些患者进行长期安全随访现在必须成为首要任务。
更新日期:2022-07-16
中文翻译:
早期治疗是 SMA 婴儿的生命线
在 3 期 SPR1NT 试验中,症状前基因治疗在基因诊断为脊髓性肌萎缩症 (SMA) 的婴儿中表现出令人印象深刻的临床结果;对这些患者进行长期安全随访现在必须成为首要任务。