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theguardian.com
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FDA Rejects Promising Drug for Rare Mitochondrial Diseases
The FDA rejected elamipretide, a drug showing promise for treating rare mitochondrial diseases like MLS syndrome, despite positive patient testimonials and an advisory committee's narrow recommendation, raising concerns about the future of drug development for ultra-rare conditions.
FDA Rejects Promising Drug for Rare Mitochondrial Diseases
The FDA rejected elamipretide, a drug showing promise for treating rare mitochondrial diseases like MLS syndrome, despite positive patient testimonials and an advisory committee's narrow recommendation, raising concerns about the future of drug development for ultra-rare conditions.
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