risdiplam

Risdiplam is an orally bioavailable small molecule mRNA splicing modifier used for the treatment of spinal muscular atrophy (SMA), a rare genetic neuromuscular disorder characterized by loss of lower motor neurons and progressive muscle wasting[1][2][5]. Risdiplam works by modifying the splicing of SMN2 pre-mRNA to promote inclusion of exon 7, resulting in increased production of full-length survival motor neuron (SMN) protein[5][6][7][8]. This compensates for the deficiency caused by mutations in the SMN1 gene. Risdiplam is approved for use in adults, children, and infants with SMA and offers oral administration as an advantage over other therapies that require intrathecal or intravenous delivery[3][4]. The drug was developed by Roche/Genentech and is marketed under the brand name Evrysdi.

Brand names

Evrysdi

Other names

risdiplam