Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome

202220 citationsJournal Articlegold Open Access

Authors

Brittany Hornby · Kennedy Krieger Institute

William Thompson · Johns Hopkins University

Mohammed Almuqbil · King Saud bin Abdulaziz University for Health Sciences

Ryan Manuel · Johns Hopkins University

Anthony Abbruscato · Stealth BioTherapeutics (United States)

Jim Carr · Stealth BioTherapeutics (United States)

Hilary J. Vernon

Abstract

A matched Natural History Control (NHC) was used to evaluate elamipretide in BTHS Elamipretide may improve natural history of BTHS by attenuating natural decline in heart function Elamipretide was associated with meaningful clinical improvements in skeletal muscle and cardiovascular parameters that were not observed in NHCs The study established a NHC for use in assessing the efficacy of therapeutic interventions in BTHS.

Topics & Keywords

Cardiac electrophysiology and arrhythmias Ion channel regulation and function Mitochondrial Function and Pathology

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Orphanet Journal of Rare Diseases

Volume 17, Issue 1, pp. 336-336

DOI: 10.1186/s13023-022-02469-5

Field-Weighted Citation Impact: 3.28