Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Thompson WR, Manuel R, Abbruscato A, et al. Genetics in Medicine 2024;26:101138.
What it found
Injection-site reactions were the most common side effect. 6-minute walk distance improved at every visit (96.1 m by week 168), fatigue scores stayed below baseline, and some heart volume measures trended better.
Limitations
No control group; 8 of 10 people reached week 168. Three authors were employees of the developer.
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