ultra-rare disease – Talk Bio

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FDA Advisory Committee Supports Stealth BioTherapeutics’ Elamipretide for Barth Syndrome Despite Data Concerns

6 months ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, Advisory Committees, Elamipretide, Stealth BioTherapeutics, ultra-rare disease, United States Food and Drug Administration, unmet

Stealth BioTherapeutics, elamipretide, Barth syndrome, FDA advisory committee, ultra-rare disease, unmet medical need

FDA Raises Concerns Over Stealth BioTherapeutics’ Ultra-Rare Disease Drug Ahead of Advisory Committee Meeting

7 months ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, Advisory Committees, concerns, Drug Approval, Effectiveness, Elamipretide, Stealth BioTherapeutics, ultra-rare disease, United States Food and Drug Administration

FDA, Stealth BioTherapeutics, elamipretide, Barth syndrome, ultra-rare disease, advisory committee meeting, drug approval, efficacy concerns