林业发展局批准了Waskyra的Wiskott-Aldrich综合症基因治疗,这是一种罕见的免疫障碍。 The FDA approved Waskyra gene therapy for Wiskott-Aldrich syndrome, a rare immune disorder.

FDA批准了针对Wiskott-Aldrich综合征的基因疗法Waskyra，而ianalumab在减少免疫性血小板减少症的出血和B细胞耗竭方面展现出希望，而bezuclastinib在非晚期系统性肥大细胞增多症中显示出显著的生物标志物降低效果。 The FDA approved the gene therapy Waskyra for Wiskott-Aldrich syndrome, while ianalumab showed promise in reducing bleeding and depleting B cells in immune thrombocytopenia, and bezuclastinib demonstrated strong biomarker reduction in non-advanced systemic mastocytosis. 2025年夏令营会议和其他最近的会议都强调了这些进展,反映了针对罕见和复杂疾病的定向治疗所取得的进展。 These advances were highlighted at the ASH 2025 meeting and other recent conferences, reflecting progress in targeted treatments for rare and complex disorders.