We are an ultra-rare community, but we demonstrated the global reach of our #BarthSTRONG family in our recent advocacy efforts in support of a new drug application for elamipretride. We want all Barth syndrome patients to have access to this potential new treatment that we think is safe and has shown positive effects during the recently completed clinical trial. With your help, we received over 4,200 signatures and 730 testimonials on our BSF Community Petition that was submitted to the U.S. Food and Drug Administration (FDA) and which can be found at www.barthsyndrome.org/petition.

We’ve heard of how the debilitating and constant fatigue experienced by our affected individuals can profoundly impact every daily activity. It is our aspiration that the improvements in energy, stamina, and quality of life in addition to the important positive changes in certain cardiac measures found in the assessment of elamipretide for Barth syndrome ultimately will translate into access for our entire community. It is heartening that, with your signatures and stories, we have an opportunity to make our collective voice heard to the FDA and Stealth BioTherapeutics.

In addition, there also has been an effort on the part of key physicians and other experts with specific knowledge about caring for those with Barth syndrome to support access to elamipretide. Twenty-six prominent experts signed a letter of support stating, in part, that “we want to have the current opportunity to prescribe elamipretide to our patients with Barth syndrome” that also has been sent to the FDA and also can be found on our website at www.barthsyndrome.org/petition. Though our advocacy has only begun, our community-wide efforts have supported our goal to get approval for the first-ever potential therapy for Barth syndrome.