Mechanism of action<unk>  addresses the underlying cause of SMA: a reduced amount of survival motor neuron (SMN) protein. The protein is encoded by the SMN1 and SMN2 genes. SMA is caused by mutations in SMN1 that code for inactive forms of the protein. The activity of the SMN2 gene, which produces much smaller quantities of SMN, tends to determine the severity of disease. The compound is a pyridazine derivative that modifies the splicing of SMN2 messenger RNA to include exon 7, resulting in an increase in the concentration of the functional SMN protein in vivo. Nusinersen, the first drug approved to treat SMA, an anti-sense oligonucleotide targeting intronic splicing silencer N1 (ISS-N1), also alters mRNA splicing of SMN2.
Risdiplam
Lemborexant
Fenproporex
Tasimelteon
Fosaprepitant
Bocasan
Mitapivat
Pleconaril