SMA drug now approved for patients of all ages

Genentech announced that the FDA approved a label extension for Evrysdi to include infants younger than 2 months with spinal muscular atrophy.
The approval is based on interim efficacy and safety data from the RAINBOWFISH study, which showed that in newborns with two or three copies of the SMN2 gene (n = 6), 100% were able to sit after 1 year of treatment with Evrysdi (risdiplam, Genentech/Roche Group), 67% were able to stand, and 50% could walk independently. All infants were alive at 12 months without permanent ventilation, according to a company press release.
“The approval of Evrysdi

Genentech announced that the FDA approved a label extension for Evrysdi to include infants younger than 2 months with spinal muscular atrophy.
The approval is based on interim efficacy and safety data from the RAINBOWFISH study, which showed that in newborns with two or three copies of the SMN2 gene (n = 6), 100% were able to sit after 1 year of treatment with Evrysdi (risdiplam, Genentech/Roche Group), 67% were able to stand, and 50% could walk independently. All infants were alive at 12 months without permanent ventilation, according to a company press release.
“The approval of Evrysdi