Barth syndrome

Ultra-rare disease

question ask wonder who why
Biotech

FDA denies request to reconsider Stealth's ultrarare disease med

The biotech says it's "evaluating all options" after the FDA directed the biotech to resubmit a new drug application for its rare disease candidate.
Gabrielle Masson Aug 8, 2025 1:58pm
FDA Adcomm

After agony, FDA adcomm favors Stealth's ultra-rare disease drug

Oct 10, 2024 6:20pm