LMI070

LMI070

CAT N°: 26757
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From 66.00 56.10

LMI070 is an SMN2 splice modulator.{42746} It selectively enhances SMN2 splicing by stabilizing the complex formed by the U1 small nuclear ribonucleic protein (snRNP) and SMN2 pre-mRNA. LMI070 (3-30 mg/kg) increases the expression of the full-length SMN2 trancript and, at doses ranging from 0.3 to 30 mg/kg, increases SMN protein levels in the brain and spinal cord in the C/+ mouse model of spinal muscular atrophy (SMA).{42746,42747} It also increases survival of SMN?7 mice, a model of severe SMA, when administered at doses of 1 and 3 mg/kg.{42746}

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