Tag: Dr Hani N Sabbah
The heart of the matter: Elamipretide in the treatment of Barth syndrome

Barth syndrome (BTHS) is an ultra-rare but debilitating genetic disease affecting cell mitochondria. High infant mortality due to cardiac complications and a weakened immune system significantly decrease life expectancy. Current medications treat symptoms but not the cause, leaving an unmet clinical need to develop effective treatments that target the underlying pathology. Dr Hani N Sabbah, Professor of Medicine at Wayne […]
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