Lv11
8 积分 2026-10-23 加入
Comparison of Nusinersen Monotherapy Versus Combination Therapy With Nusinersen and Onasemnogene Abeparvovec in Spinal Muscular Atrophy Type 1 Patients With Two SMN2 Copies: A Multicenter Study From Türkiye
54分钟前
已完结
Low-dose AAV9-SMN1 with CNS-selective expression delivers efficacy and favorable safety in spinal muscular atrophy
1小时前
已完结
Incidence, Timing and Clinical Significance of Immune-Mediated Myocardial Injury and Myocarditis After Gene Replacement Therapy: A Systematic Review and Meta-Analysis
1小时前
已完结
Characterization of Safety Signals for Spinal Muscular Atrophy Treatments in Children Aged 0 to 3 Years Potential Safety Patterns for Further Investigation
1小时前
待确认
Genetic susceptibility to thrombotic microangiopathy in a 10-month-old patient with SMA type 1: a caveat for gene therapy eligibility
1小时前
已完结
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