Quality of life in Barth syndrome

por Kim, Alexander Y., Vernon, Hilary, Manuel, Ryan, Almuqbil, Mohammed, Hornby, Brittany
Publicado 2022

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 cardi...

por Reid Thompson, W., Hornby, Brittany, Manuel, Ryan, Bradley, Elena, Laux, Janice, Carr, Jim, Vernon, Hilary J.
Publicado 2020

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

por Hornby, Brittany, Thompson, William Reid, Almuqbil, Mohammed, Manuel, Ryan, Abbruscato, Anthony, Carr, Jim, Vernon, Hilary J.
Publicado 2022