Parents of children with Barth syndrome are urging the FDA to approve Elamipretide, a drug that shows promise in treating this rare and fatal disease. Current options are limited, and the denial of approval leaves families in a difficult situation.

Editor’s Note: This situation highlights the urgent need for effective treatments for rare diseases. The FDA's decision impacts not only the children suffering from Barth syndrome but also raises questions about the approval process for drugs targeting ultrarare conditions.