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28 积分 2025-09-08 加入
[Clinical practice guidelines for spinal muscular atrophy]
15小时前
已完结
Clinical practice guidelines for the care of girls and women with Turner syndrome
1个月前
已完结
Turner syndrome: mechanisms and management
2个月前
已完结
Gene therapy for inborn error of immunity – current status and future perspectives
2个月前
已完结
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome
2个月前
已完结
Pathological events in platelets of Wiskott‐Aldrich syndrome patients
2个月前
已完结
Wiskott-Aldrich Syndrome
2个月前
已关闭
How I treat Wiskott-Aldrich syndrome
2个月前
已完结
A Prospective, Observational, Non-interventional Clinical Study of Participants With Choroideremia: The NIGHT Study
6个月前
已关闭
Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
6个月前
已完结