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 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.