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Time is running out for kids with a rare disease, parents say, unless the FDA acts

After a more than decadelong process to bring the experimental drug elamipretide to market, the treatment’s approval has repeatedly hit roadblocks.

by NBC News | August 18, 2025
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Dr. Kathryn Chatfield, Gilbert’s doctor and a specialist in pediatric cardiology and genetics at Children’s Hospital Colorado, said the FDA’s inaction leaves families with few alternatives.