PTC258 is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases the expression of ELP1 in vitro and in vivo. PTC258 is well tolerated in mouse model.
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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