Sign our petition for a fair, equitable and appropriate review of elamipretide in Barth syndrome by the FDA before we lose access to this drug, the only medicine currently in development for our life-threatening, ultra-rare disease. Together, we are #BarthSTRONG and there is power in numbers. Our goal is to get 10,000 signatures and we know that we can do it with the power this community has.
Not only is your signature helping the Barth syndrome community, but the thousands of other ultra-rare diseases without FDA approved treatments!
We would also like to thank Declan's family for sharing their story and what is at stake if access to elamipretide is lost. Less than three months after starting elamipretide, Declan’s heart structure and function improved more than doctors ever expected. Declan also regained strength and energy. Now, after eight months on this treatment, Declan has had the ventricular assist device removed (without requiring a heart transplant), and he was recently discharged from the hospital. His doctors said that they are not aware of any other patient with Barth syndrome who was able to transition off a ventricular assist device without requiring a heart transplant. His doctors credit elamipretide with making the difference.
Sign the petition here.
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