Barth syndrome is a rare, life-threatening disease causing heart failure, muscle weakness, and infections, tragically taking too many young lives. With your support, we are making meaningful progress and helping those affected by Barth syndrome.
Advocacy and Awareness
- Historic FDA Advocacy: Amplifying our community’s voice during the ongoing FDA review of elamipretide, bringing us closer to the first-ever approved therapy.
- Amplifying Voices: Advocates meet with lawmakers during Rare Disease Week to ensure the voices of Barth syndrome families are heard in the halls of government.
Family Support
- Supporting Our Barth Families: The Emergency Relief Program helps families with unexpected expenses, allowing them to focus on care.
- Connecting Families at the International Conference: The Family Services Fund provides financial support for families to connect with experts and each other.
- Breaking Language Barriers: Translation services ensure all families can engage in vital programs.
Research and Progress
- Advancing Research: Donations fuel studies that bring us closer to transformative therapies and a cure.
- Expanding the Barth Syndrome Registry (BRR): Contributions to the BRR help identify key symptoms, like gastrointestinal issues, as diagnostic markers.
Our Shared Vision: Progress That Powers Hope
Built on over two decades of dedication, your support ensures a brighter future for those impacted by Barth syndrome.
Every donation counts. Your commitment is the #GiftofPROGRESS, fueling hope for tomorrow. Thank you for your support.
You can learn more about the Barth Syndrome Foundation by visiting: barthsyndrome.org