Tipo di evidenza
Studio randomizzato sull’uomo
SS-31 component long-term human evidence
Pubblicazione e risultato principale
Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER.
During a 168-week open-label extension in Barth syndrome, elamipretide was associated with a cumulative 96.1-metre improvement in six-minute walk distance, lower fatigue scores and improved cardiac-volume measures.
Genetics in medicine : official journal of the American College of Medical Genetics


