Risdiplam

Risdiplam (RG7916, RO7034067; Evrysdi is a novel, potent, orally bioactive, central and peripherally distributed gene splicing modulator (neuromuscular disease) approved for the treatment of spinal muscular atrophy. Risdiplam increases survival motor neuron (SMN) protein levels.
Risdiplam increases the amount of SMN protein and controls splicing of pre-SMN2 mRNA to generate full-length SMN2 mRNA. Risdiplam is a splicing modulator of SMN2, which can increase the amount of full-length SMN2 protein, thereby enhancing the function of SMN protein. The most common genetic disease that causes infant death remains SMA (SMA). Low levels of early motor neuron protein (SMN), due to a duplex in the Southeast motor neuron 1 (SMN1) gene, are responsible for this autosomal recessive neuropathy, which is characterized by progressive weakening of motor and respiratory muscles. Residual inactivation and other sources of gene loss