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In:
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Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy. [electronic resource] by
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In:
Journal of medicinal chemistry vol. 60
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Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 exon 7. [electronic resource] by
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In:
Genomics vol. 105
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Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA). [electronic resource] by
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- Green, Luke
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- Jablonski, Philippe
- Jacobsen, Bjoern
- Khwaja, Omar
- Kletzl, Heidemarie
- Ko, Chien-Ping
- Kustermann, Stefan
- Marquet, Anne
- Metzger, Friedrich
- Mueller, Barbara
- Naryshkin, Nikolai A
- Paushkin, Sergey V
- Pinard, Emmanuel
- Poirier, Agnès
- Reutlinger, Michael
- Weetall, Marla
- Zeller, Andreas
- Zhao, Xin
- Mueller, Lutz
Producer: 20190618
In:
Journal of medicinal chemistry vol. 61
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Revised Recommendations for the Treatment of Infants Diagnosed with Spinal Muscular Atrophy Via Newborn Screening Who Have 4 Copies of SMN2. [electronic resource] by
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- Darras, Basil T
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- Kuntz, Nancy
- Prior, Thomas
- Shieh, Perry B
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Producer: 20201102
In:
Journal of neuromuscular diseases vol. 7
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