Elamipretide

Unassigned

New Medicines

Barth syndrome

Information

New molecular entity
Stealth BioTherapeutics
Stealth BioTherapeutics

Development and Regulatory status

None
None
Phase III Clinical Trials
Yes

Category

Mitochondrial permeability transition pore inhibitor
Barth syndrome is a rare, metabolic, and neuromuscular, genetic disorder that occurs exclusively in males. Primary characteristics include cardioskeletal myopathy, neutropenia and growth retardation potentially leading to short stature [3,4]. Epidemiologists in the US estimate the incidence of Barth syndrome to be between 1 in 200,000 to 1 in 400,000 births. These figures may be underestimated as many cases go undiagnosed [1].
Barth syndrome
Subcutaneous