Tipo di evidenza
Studio randomizzato sull’uomo
Research relevant to SS-31
Pubblicazione e risultato principale
Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER.
The study reports that mean BTHS-SA Total Fatigue scores were below baseline (improved) at all OLE time points.
Genetics in medicine : official journal of the American College of Medical Genetics



