DOI:
10.1055/s-00000071
Seminars in Neurology
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References
Kletzl H, Marquet A, Günther A. , et al.
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.
Neuromuscul Disord 2019;
29 (01) 21-29
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