"My wish is to live one day normal without having Barth Syndrome," Henry said...
CBS Bay Area came and interviewed our family in June 2025 about the disappointing news we received from the FDA about elamipretide. Check out the news clip here, and be sure to read the transcript below the clip as it explains the issue a bit better. Hopeful that our story will help create more awareness around what has happened, and potentially impact the FDA's next steps.
The message remains the same...the FDA has all of the data on efficacy and safety they need to make a decision. No concerns have been raised about either. Real people are having measurable life-changing results that show elamipretide is positively treating Barth syndrome. The Barth syndrome community's experience with this process is a litmus test. If something this promising—backed by strong science, expert clinicians, hopeful and engaged families, and even a positive advisory committee vote—can’t move forward, the future of all rare disease research and drug development is on the line.
Our story is one of many. It is not over yet ~ please share this story in hopes the FDA will make this right.
The message remains the same...the FDA has all of the data on efficacy and safety they need to make a decision. No concerns have been raised about either. Real people are having measurable life-changing results that show elamipretide is positively treating Barth syndrome. The Barth syndrome community's experience with this process is a litmus test. If something this promising—backed by strong science, expert clinicians, hopeful and engaged families, and even a positive advisory committee vote—can’t move forward, the future of all rare disease research and drug development is on the line.
Our story is one of many. It is not over yet ~ please share this story in hopes the FDA will make this right.
