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 Heuberger, Geert Jan Groeneveld, Willem Birkhoff, Eugenio Mercuri, Hanns Lochmüller, Claire Wood, Dirk Fischer, Irene Gerlach, Katja Heinig, Teodorica Bugawan, Sebastian Dziadek, Russell Kinch, Christian Czech, Omar Khwaja

Research output: Contribution to journal › Article › peer-review

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Medicine & Life Sciences