The Barth Syndrome Community Needs Action Now

Get Involved in Advocacy for Barth Syndrome

Those with Barth syndrome deserve more from the FDA. We are asking for a fair, equitable and appropriate review of an investigational drug, called elamipretide, by the FDA before patients lose access to this drug. It is the only medicine currently in late-stage development for our life-threatening, ultra-rare disease, Barth syndrome.

On behalf of the entire Barth Syndrome Foundation community, please help us ask the FDA to incorporate the patient’s perspective and fully review all the data now.

Get Involved By Sharing Your Story

Voice Your Support Write to the FDA

Get in Touch

If you are interested in further getting involved by sharing your story with the media or attending an advocacy meeting, please let us know by completing the form below. Please use this form to also submit any media inquiries.