利司扑兰口服溶液用散 (艾满欣®) 60mg

Description

Risdiplam is a survival motor neuron 2 (SMN2) splicing modifier.
It is the first oral medication approved to treat Spinal Muscular Atrophy (SMA) in pediatric and adult patients.
It works by increasing and sustaining the production of the SMN protein throughout the central nervous system and peripheral tissues, which is critical for maintaining motor neurons and muscle function.

利司扑兰是一种脊髓性肌萎缩症 (SMA) 治疗药物，属于生存运动神经元 2 (SMN2) 基因剪接修饰剂。它是首个获批用于治疗儿童和成人 SMA 的口服药物。它通过增加并维持中枢神经系统和外周组织中 SMN 蛋白的产生来发挥作用，这对于维持运动神经元和肌肉功能至关重要。

Dosage Form: Powder for oral solution
Concentration: 60mg per bottle (每瓶含利司扑兰 60mg).
Manufacturer: Roche / Genentech (罗氏).