SKY-0515, an investigational oral RNA splicing modifier, reduced mutant huntingtin (mHTT) protein levels in blood by up to 69% and PMS1 mRNA by up to 26% in a phase 1/2 Huntington’s disease (HD) trial.
SKY-0515, an investigational oral RNA splicing modifier, reduced mutant huntingtin (mHTT) protein levels in blood by up to 69% and PMS1 mRNA by up to 26% in a phase 1/2 Huntington’s disease (HD) trial.
SKY-0515, an investigational...