The SUNFISH Trial of Risdiplam in Type 2 and Type 3 SMA
Tom Crawford, MD, examines data from the SUNFISH trial of patients with type 2 and type 3 SMA and emphasizes the need for further data before risdiplam can be directly compared to other treatment options.
The FIREFISH Trial of Risdiplam in Type 1 SMA
Basil Darras, MD discusses the FIREFISH trial of risdiplam in infants with type 1 symptomatic SMA and comments on the difficulty of comparing agents when trials have had different enrollment criteria.
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.