Efficacy of Risdiplam in Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis.
Carlos Pascual-MorenaVicente Martínez-VizcaínoIván Cavero-RedondoIrene Martínez-GarcíaNerea Moreno-HerráizCelia Álvarez-BuenoAlicia Saz-LaraPublished in: Pharmacotherapy (2023)
The limited available evidence suggests that risdiplam is an effective and safe drug for the treatment of SMA. In addition, long-term clinical benefit may be partly determined by the stage of disease at which treatment is initiated.