ML372 inhibits survival motor neuron ( SMN ) protein ubiquitination, increases SMN protein stability without affecting mRNA expression. ML372 improves spinal muscular atrophy (SMA) in mice. ML372 is brain penetrant and has a reasonable exposure and half-life in vivo.
Products | Products > Disease Areas > Neurological, Eye or Ear Disease | Products > Signaling Pathways > Cell Cycle/DNA Damage | Products > Research Areas > Neurological Disease | Products > Signaling Pathways > DNA/RNA Synthesis
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