SOURCE-LINKED STUDY SUMMARY
Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy.
Abstract summarizedQuality not appraised
- What was studied
- A randomized, double-blind trial compared intravenous elamipretide with placebo. The primary outcome was change in six-minute walking distance after five days.
- Who / model studied
- The study included 36 adults with genetically confirmed primary mitochondrial myopathy.
- Studied dose & schedule
- Dose and schedule have not yet been extracted for this summary.
- What the study found
- Walking distance increased by 64.5 metres at the highest dose versus 20.4 metres with placebo; this unadjusted comparison did not reach statistical significance. Dose-response and adjusted analyses favored elamipretide. Other efficacy and safety outcomes showed no significant differences.
- Limitations & context
- The authors called for larger trials with longer treatment periods. These five-day results do not establish sustained benefit or long-term safety.
Read summary source ↗ · Prepared 9/22/2026 · AI-assisted, source-based summary
Review record & corrections
Summary prepared: . Source scope: abstract. This is an AI-assisted source summary, not an independent clinician sign-off.
No public field-by-field revision history is available for this card yet. The date above is the summary date, not proof that every field was updated then.
Inspect the source · Correction policyResearch findings apply to the population and conditions studied. This educational summary is not a treatment recommendation or a formal assessment of study quality.
Original publication ↗ · DOI: 10.1212/wnl.0000000000005255