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Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of

Hasane Ratni1, Martin Ebeling1, John Baird2

  • 1F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.

Journal of Medicinal Chemistry
|July 26, 2018
PubMed

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