An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

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Reenie McCarthy, CEO of Stealth Biotherapeutics, discusses the challenges the company has faced in seeking FDA approval for its experimental therapy to treat the rare and life-threatening condition Barth syndrome, the lack of consistency the company found from division to division within the agency, and why this could have a chilling effect on the development of ultra-rare disease therapies if left unaddressed.