O07: Optimization of positive investigational outcomes in Barth syndrome using a combination of placebo-controlled, extended open-label, and natural history comparison studies*

• Hilary Vernon,
• Brittany Hornby,
• Anthony Abbruscato,
• Jim Carr,
• W. Reid Thompson

Affiliations

Hilary Vernon

Kennedy Krieger Institute

Brittany Hornby

Kennedy Krieger Institute

Anthony Abbruscato

Stealth Biotherapeutics, Inc

Jim Carr

Stealth Biotherapeutics, Inc

W. Reid Thompson

Johns Hopkins University School of Medicine