TrialGenetics in medicine : official journal of the American College of Medical Genetics2021

A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism.

W Reid Thompson et al.PubMed ↗Full text ↗Publisher ↗

No numbers read from the abstract.