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