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306 积分 2025-08-04 加入
Building safeguards for gene therapy in haemophilia
12天前
已完结
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy
26天前
已完结
Identifying Parameters Associated with Delayed Diagnosis in Thrombotic Antiphospholipid Syndrome: Data from China Prospective APS Cohort
1个月前
已完结
Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance
2个月前
已关闭
Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study
4个月前
已完结
Huntington’s Disease: Latest Frontiers in Therapeutics
5个月前
已完结
What Is Huntington Disease?
5个月前
已完结
Huntington’s disease treated for first time using gene therapy
5个月前
已完结
Safety and efficacy of a novel ANGPTL4 inhibitory antibody for lipid lowering: results from phase 1 and phase 1b/2a clinical studies
7个月前
已完结
GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant
8个月前
已完结
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