Barth Syndrome Awareness Day is celebrated around the world on April 5th to bring together all members of our ultra-rare disease community for a single cause: To increase awareness of Barth syndrome so all affected individuals can access safe and effective treatments. Barth syndrome is caused by a mutation in the tafazzin gene, also called G4.5. For this reason, 4/5 (or April 5th in the United States) is the designated day around which individuals and their families highlight the need for accurate and early diagnosis, advancements in research and development, and access to therapies. With the first potential new drug for Barth syndrome so close to regulatory approval, now more than ever we celebrate this day as a call-to-action to make therapies possible and accessible for people with Barth syndrome. Read the full press release.
If you have Barth syndrome or know someone who does, we encourage you to participate in Barth Syndrome Awareness Day by clicking any or all of the following ways: