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ISIS-SMNRx

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Company Isis Pharmaceuticals Inc.
DescriptionAntisense oligonucleotide that modulates splicing of survival of motor neuron 2 centromeric (SMN2) mRNA
Molecular Target Survival of motor neuron 2 centromeric (SMN2)
Mechanism of ActionAntisense therapy
Latest Stage of DevelopmentPhase II
IndicationSpinal muscular atrophy (SMA)
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