Let's get elamipretide FDA approved

Hi everyone,

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This is our baby boy Gilbert. Shortly after he was born, Gilbert was diagnosed with a very rare genetic condition called Barth syndrome. Due to heart failure, we were in the hospital cardiac care unit with Gilbert for 80 days. The main reason he’s home right now is because of a new drug called elamipretide (pronounced “EL-EM-EP-REE-tide”).

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On May 29, the FDA decided not to approve elamipretide. While we are a newly diagnosed family, the journey to try and get elamipretide FDA approval is not new. This process has taken years. Our family, and the families of other boys affected by Barth syndrome, needs community support in getting the FDA to reverse its decision and approve elamipretide for all who need it—especially infants in critical need, like Gilbert.

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There is no doubt in our mind that elamipretide saved Gilbert’s life. There are no major side effects for elamipretide, and there is such a huge upside for patients like him. We need full access now.

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We are afraid that if elamipretide is not approved in the next 6-9 months, Gilbert will lose access to this life-saving drug.

 

If you can help us draw more attention to this issue we would greatly appreciate it.

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This is an issue that extends beyond the Barth syndrome community. All rare diseases need a better pathway for approval of new, promising treatments.

 

Thank you for your support!