怀孕早期暴露于帕蒂西兰:一个案例报告
Valentin Loser1, Thomas Baumgartner2, Hélène Legardeur3
1Service of Neurology, Department of Clinical Neurosciences, Lausanne University Hospital, University of Lausanne, Rue du Bugnon 44, Lausanne 1011, Switzerland.
Therapeutic advances in neurological disorders
|March 27, 2024
概括
本案例研究报告了第一次怀孕暴露于帕蒂西兰 (小干扰RNA) 遗传性跨甲基素相关的粉症. 怀孕过程没有任何严重的胎儿并发症,尽管母亲的维生素A水平需要谨慎管理.
科学领域:
- 抗RNA干扰疗法RNA干扰疗法药物
- 怀孕在罕见疾病中的结果.
- 神经病的管理神经病的管理.
背景情况:
- 遗传性转基因蛋白相关的粉症 (hATTR粉症) 是一种渐进的多系统性疾病.
- 小干扰RNA (siRNA) 的帕蒂西兰是一种有效的hATTR氨基粉症治疗方法,但其在怀孕期间的安全性尚不清楚.
- 在怀孕期间管理慢性疾病需要平衡治疗疗效与潜在的胎儿风险.
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