Efficacy of Risdiplam in Spinal Muscular Atrophy: A Systematic Review and Meta ‐Analysis

ConclusionThe 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.

Source: Pharmacotherapy: The Journal of Human Pharmacology and Drug Therapy - August 14, 2023 Category: Drugs & Pharmacology Authors: Carlos Pascual ‐Morena, Vicente Martínez‐Vizcaíno, Iván Cavero‐Redondo, Irene Martínez‐García, Nerea Moreno‐Herráiz, Celia Álvarez‐Bueno, Alicia Saz‐Lara Tags: REVIEW Source Type: research