Risdiplam for the Treatment of SMA
Expert pediatric neurologist Carolina Tesi Rocha, MD gives insight into the mechanism of action of risdiplam and comments on the need for further monitoring of its potential adverse effects.
The SPR1NT Trial of Onasemnogene Abeparvovec in SMA
Claudia Chiriboga, MD, MPH reviews data from the SPR1NT clinical trial of patients with presymptomatic SMA and emphasizes how the trial highlights the importance of prenatal screening and early treatment.