Walker Burger is beside himself with worry.
The 33-year-old relies on an experimental medicine to treat an ultra-rare disease called Barth syndrome that causes an enlarged heart, muscle weakness, and a shortened life expectancy. But the Food and Drug Administration last year refused to review the drug, despite prolonged efforts by its manufacturer, Stealth BioTherapeutics, to gather hard-to-come-by clinical data on such a tiny patient population.
As a result, Burger, who participated in a small clinical trial and continues to receive the drug through an access program, is concerned regulatory approval may never come — and his supply will disappear.
This article is exclusive to STAT+ subscribers
Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts.
Already have an account? Log in
About the Author Reprints
To submit a correction request, please visit our Contact Us page.
