DNA/RNA Synthesis

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  1. SMN2 splicing modulator

    SMN-C3 is an orally active SMN2 splicing modulator and has the potential to treat spinal muscular atrophy (SMA).
  2. SMN2 splicing modulator

    Branaplam (LMI070) is a highly potent, selective and orally active small molecule SMN2 splicing modulator.

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