Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

"Risdiplam is administered orally once daily and the recommended dosage is determined by age and body weight (see Table 1) \[as outlined in the US Prescribing Information for EVRYSDI™(risdiplam)\].~Table 1 Adult and Pediatric Dosing Regimen by Age and Body Weight: US Prescribing Information~Age and Body Weight (Recommended Daily Dosage): 2 months to less than 2 years of age (0.2 mg/kg); 2 years of age and older weighing less than 20 kg (0.25 mg/kg); 2 years of age and older weighing 20 kg or more (5 mg)"