Barth syndrome

Ultra-rare disease

accelerated
Biotech

FDA aims for September approval decision for Stealth's drug

The newfound urgency is likely fueled by a public outcry surrounding the FDA's several delays and subsequent refusal of the rare disease candidate.
Gabrielle Masson Aug 21, 2025 10:15am
question ask wonder who why

FDA denies request to reconsider Stealth's ultrarare disease med

Aug 8, 2025 1:58pm
FDA Adcomm

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

Oct 10, 2024 6:20pm