Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Thompson WR et al. · Genetics in medicine : official journal of the American College of Medical Genetics · 2024
The study reportedElamipretide was associated with sustained long-term tolerability and efficacy, with improvements in functional assessments and cardiac function in BTHS.