Risdiplam 是一种中枢和外周分布的 SMN2 前 mRNA 剪接修饰剂，可增加运动神经元存活 (SMN) 蛋白水平。

Risdiplam (RG7916) is orally administered. Risdiplam is a centrally and peripherally distributed SMN2 pre-mRNA splicing modifier which increases survival motor neuron (SMN) protein levels[1].

Risdiplam is a modifier of SMN2 splicing, leading to an increase in SMN2 full-length transcript and thus functional SMN protein. Risdiplam modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA and increases SMN protein levels [1]. Spinal muscular atrophy (SMA) type I remains the most common genetic disease resulting in death in infancy. Characterized by progressive motor and respiratory muscle weakness, this autosomal recessive neuromuscular disorder is caused by low levels of the survival motor neuron protein (SMN) due to inactivating bi-allelic deletions and other disabling mutations in the survival motor neuron 1 (SMN1) gene[1]. Risdiplam is a modifier of SMN2 splicing, leading to an increase in SMN2 full-length transcript and thus functional SMN protein. Risdiplam modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA and increases SMN protein levels [1]. Spinal muscular atrophy (SMA) type I remains the most common genetic disease resulting in death in infancy. Characterized by progressive motor and respiratory muscle weakness, this autosomal recessive neuromuscular disorder is caused by low levels of the survival motor neuron protein (SMN) due to inactivating bi-allelic deletions and other disabling mutations in the survival motor neuron 1 (SMN1) gene[1].

1825352-65-5

C22H23N7O

401.46

RG7916;RO7034067

Ethanol:2 mg/mL (4.98 mM),Need ultrasonic
DMSO:2 mg/mL (4.98 mM,Need ultrasonic
H2O:2.22 mg/mL (5.53 mM),ultrasonic and adjust pH to 6 with HCl
Powder: -20°C for 3 years
In solvent: -80°C for 2 years