Medical Xpress
From Johns Hopkins University School of Medicine, Charlotte J. Sumner, M.D., presents an editorial on a study by Richard S. Finkel and colleagues, who report an open-label, Phase II trial of the pre-messenger RNA splicing modifier risdiplam in presymptomatic spinal muscular atrophy…
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Open-label Phase II trial reports early motor milestones with risdiplam
Tyisha Volkman
03:21
From Johns Hopkins University School of Medicine, Charlotte J. Sumner, M.D., presents an editorial on a study by Richard S. Finkel and colleagues, who report an open-label, Phase II trial of the pre-messenger RNA splicing modifier risdiplam in presymptomatic spinal muscular atrophy…
