Study design and objectives
BTHS
The current study focused on examining the collected data to detect hidden structure in physiological measurements as a means of unfolding and thus describing heterogeneity in treatment response to elamipretide in patients with BTHS. For this purpose, the study first assessed whether features extracted from time series data of each of the 3 study visits (Base1, End1, and End2) could classify patients according to functional status (“highest score” and “lowest score”). Secondly, we applied hierarchical clustering to select features that can adequately distinguish “responders” from “non-responders” to elamipretide.