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2018•Neuromuscular DisordersRequires access

The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy

Heidemarie Kletzl, Anne Marquet, Andreas Günther, Wakana Tang, Jules A. A. C. Heuberger, Geert Jan Groeneveld, Willem Birkhoff, Eugenio Mercuri, Hanns Lochmüller, Claire Wood, Dirk Fischer, Irene Gerlach, Katja Heinig, Teodorica L. Bugawan, Sebastian Dziadek, Russell Kinch, Christian Czech, Omar Khwaja

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Key concepts: SMN1, Spinal muscular atrophy, SMA*, Motor neuron, Exon, Alternative splicing, Biology, RNA splicing

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The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy — Research Paper | ScholarLens