Rankin County teen wins battle for treatment with FDA drug approval WLBTFDA approves ultra-rare disease drug that became a symbol of uncertainty over regulatory standards statnews.comFDA greenlights Stealth Bio’s injection as 1st treatment for Barth syndrome Fierce PharmaStealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY™ (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome PR NewswireFDA approves Stealth’s rare disease drug after past rejection, delay Endpoints News
