Lv1
50 积分 2025-01-10 加入
Dose-finding and optimization in drug development for rare diseases
4个月前
已完结
Low-dose AAV9-SMN1 with CNS-selective expression delivers efficacy and favorable safety in spinal muscular atrophy
4个月前
已完结
Can NRF2 provide a gene-agnostic approach to treating age-related macular degeneration?
4个月前
已完结
Engineering immune tolerance for AAV gene therapy: The Norse way
4个月前
已完结
Randomized phase 2b dose-escalation trial of stem cell therapy with laromestrocel for aging frailty
5个月前
已完结
Developmental and epileptic encephalopathies
5个月前
已完结
Dravet syndrome diagnosed in adults
5个月前
已完结
Safety and efficacy of hemobeglogene autotemcel(hemo-cel) gene therapy in five patients with transfusion-dependent β-thalassemia
5个月前
已完结
Hemophilia A gene therapy: Moving to the next chapter
5个月前
已完结
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