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Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam

A study of risdiplam in infants with presymptomatic spinal muscular atrophy (SMA) Kathryn Wagner, M.D. Ph.D., Vice President of Neuromuscular Disorders Clinical Development

Kathryn Wagner, M.D. Ph.D., Vice President of Neuromuscular Disorders Clinical Development Paulo Fontoura, M.D. Ph.D., Global Head of Neuroscience, Immunology, Ophthalmology, Infectious and Rare Diseases Clinical Development Simona Skerjanec, Therapeutic Area Head Neuroscience and Rare Diseases, Global Product Strategy Kavita Patel, Lifecycle Leader SMA, Global Product Strategy