CO29 Real-World Benefit of Ataluren Treatment in Milestones Not Related to Ambulatory Function in Nonsense Duchenne Muscular Dystrophy Versus Standard of Care

To present the most recent data on real-world ataluren benefits on upper limb and pulmonary function, compared to standard of care (SoC).

Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: D. Machado, E. Paula, I.D.L. Bretas, A.L.M. Freitas, A.P.C. dos Santos Source Type: research