With nearly 250 individuals already registered and eager to participate, we invite you to be a part of this enriching conference. Our program is thoughtfully designed to focus beyond symptoms of Barth syndrome, offering practical tools and insights for those living with or caring for a loved one with Barth syndrome. Reconnect with familiar faces and forge new connections as we come together to learn, grow, and support one another.
REGISTRATION IS OPEN! Register for the conference now through the link provided below. Experience the convenience of our new conference app, enabling you to personalize your agenda like never before. Upon registration, simply download the app and begin crafting your tailored agenda right away.
The Barth Syndrome Foundation (BSF), the only patient advocacy organization dedicated to Barth syndrome and saving lives around the world through education, advances in treatments and finding a cure, today recognizes the second annual Barth Syndrome Awareness Day. Barth syndrome is an ultra-rare, life-threatening, genetic disease primarily affecting males. Currently, there are no FDA-approved treatments. The FDA's recent decision to review a New Drug Application (NDA) for elamipretide in Barth syndrome represents a significant step forward in addressing the critical unmet needs of patients suffering from this devastating, progressive condition.
Boston, April 3, 2024 - We have learned from Stealth BioTherapeutics that the U.S. Food and Drug Administration (FDA) has agreed to file and review the new drug application (NDA) for elamipretide for the treatment of Barth syndrome. The NDA, submitted by the drug’s sponsor Stealth BioTherapeutics, is a crucial step in BSF’s journey to advance treatments and find a cure for Barth syndrome. We are encouraged by this news!
We are excited to announce that Lindsay T. Marjoram, PhD, will be joining BSF as a senior staff member on Nov 6, 2023!
Congratulations to Melissa Huang, PhD on being promoted to BSF's Research Associate!
Sign our petition for a fair, equitable and appropriate review of elamipretide in Barth syndrome by the FDA before we lose access to this drug, the only medicine currently in development for our life-threatening, ultra-rare disease. Together, we are #BarthSTRONG and there is power in numbers. Our goal is to get 10,000 signatures and we know that we can do it with the power this community has.
Attend our 3-part elamipretide advocacy series to learn the importance of meeting with your legislator to ensure that elamipretide remains available.
In loving memory of Steven David Woodward (1974-2023), uncle of Connor Woodward who is affected by Barth syndrome, this BSF restricted fund, nicknamed “The 31 Fund” after Steven’s lifetime hockey number of 31, has been created to help mitigate financial challenges that would prevent certain Barth syndrome-affected individuals and families from fully participating in BSF programs.
BSF is thrilled to announce the creation of the Iris L. Gonzalez Prize (“Prize”) to advance our collective understanding of genetic variants of Barth syndrome. Generously supported by the Paula and Woody Varner Fund, BSF will award a $10,000 prize to an individual or team proposing innovative...
Please help us welcome Jonathan Stokes to the BSF Board of Directors. Jonathan is a Senior Director in Patient-Centered Outcomes Research overseeing neurology, ophthalmology, and specialty therapeutic areas at AbbVie. With 18 years of experience conducting...
After nine years of service, Kevin Woodward retires from the BSF Board in April, having served his Board limit of three consecutive terms. Kevin has been a loyal and involved Board member, not only taking on Board duties but also volunteering to be BSF’s Treasurer. Using his corporate...
Recently, Lynda Sedefian, parent of two sons with Barth syndrome, Erik and Derek, met with Rep. Paul Tonko [D-NY-20] to discuss his support of the Barth Syndrome Awareness Day Resolution in the US House of Representatives (H.Res.276). Lynda shared her experience as a mother of two individuals with Barth syndrome, the impacts of the genetic disease, and...
October is Health Literacy Month, and BSF is committed to support affected individuals and their families in expanding their health literacy. “Personal health literacy is the degree to which individuals have the ability to find, understand, and use information and services to inform health-related decisions and actions for themselves and others,” (CDC, 2020). The focus is on using health information rather than...
Disability is often viewed as a condition observable at a distance without any interaction, like someone using a wheelchair, a cane, or other assistive devices. Out of the 26 million Americans who have a severe disability, only 26% use an assistive device. The remaining majority have very little or no visual indications of disability, requiring intentional disclosure to bring awareness of their disabilities.
Conversations around disability are necessary to not only advance access and inclusion for people with disabilities, like Barth syndrome, but empower...
The Barth Syndrome Foundation (BSF), including BSF representatives as well as key clinical disease leaders, held an important workshop with Dr. Norman Stockbridge (Director of the Division of Cardiology and Nephrology in CDER at the FDA) and over 30 other representatives of different FDA centers, offices, and divisions on July 29, 2022. The stated purpose of this workshop was...
Like the falling leaves in September each year, many Barth parents visit their sons’ teachers and peers to explain what it is like to live with Barth Syndrome. BSF Board Member, Florence Mannes, is one of those parents. However, as her son, Raphaël, got older, he expressed interest in explaining Barth syndrome himself to his peers, but he wanted some...
