Thompson 2024 — TAZPOWER 168-Week Open-Label Elamipretide Extension

Genetics in Medicine

W. Reid Thompson, et al.

Summary

Long-term elamipretide was associated with sustained tolerability and functional/cardiac signals in Barth syndrome.

Study Details

Study Design

168-week open-label extension

Indication

Barth syndrome

Intervention

Long-term elamipretide

Species

Human

Risk of Bias Assessment

Open-label extension; rare disease sample