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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
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentPhase II
Standard IndicationSpinal muscular atrophy (SMA)
Indication DetailsSurvival of motor neuron (SMN)-associated spinal muscular atrophy (SMA); Treat infantile-onset spinal muscular atrophy (SMA); Treat spinal muscular atrophy (SMA)
Regulatory Designation

U.S. - Fast Track (Treat spinal muscular atrophy (SMA));
U.S. - Orphan Drug (Treat spinal muscular atrophy (SMA));
EU - Orphan Drug (Survival of motor neuron (SMN)-associated spinal muscular atrophy (SMA))

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