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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

Amyotrophic Lateral SclerosisGeneticsDegenerative DiseaseSpinal Muscular AtrophyNeurologyMotor Neuron 2MedicineSplicing VariantRna ProcessingFull Length Survival