Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome
Hornby B, Thompson WR, Almuqbil M, et al. Orphanet Journal of Rare Diseases 2022;17:336.
What it found
Compared 8 treated patients from the TAZPOWER extension with 19 untreated patients from records. Treated patients did better on walking distance (about 91 m difference at week 76) and muscle strength, and heart stroke volume rose in treated patients while it fell in untreated ones.
Limitations
Retrospective comparison with an external control group, not a randomised trial. Very small numbers. Developer employees among the authors.
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