Fig. 3
From: Oral splicing modulator branaplam in Huntington’s disease: a phase 2 randomized controlled trial
Predicted branaplam PK and PD between weeks 49 and 53 for different dosing regimens. a, Branaplam AUC. Boxes show the median and interquartile range (IQR), the whiskers extend to 1.5× IQR and the dots represent the values beyond the whiskers. The dashed horizontal line represents the median AUC between weeks 49 and 53 for the 56 mg once a 1 week treatment. Simulations performed assuming 1,000 participants per treatment group, all with the same body weight (70 kg). b, Simulated median CSF mHTT percentage ratio to baseline. The solid horizontal line shows the target decrease of 30%, the dashed horizontal line represents the mHTT 100% ratio to baseline values. Q1W, once every 1 week; Q2W, once every 2 weeks; Q4W, once every 4 weeks.
