The Barth Syndrome Foundation is thrilled to announce it has been awarded a grant from Cycle 3 of the Chan Zuckerberg Initiative’s (CZI) Rare As One Project. The CZI Rare As One Project supports and unites patient-led organizations working to advance the rare disease field by strengthening communities, building capacity, and promoting collaboration.
The 10-6 vote to recommend approval signals the advisory committee panel's assessment on elamipretide’s safety and effectiveness with its proposed intended use in Barth syndrome. The committee offers valuable perspective and non-binding recommendations for the FDA to factor in alongside other considerations during approval decisions. The FDA is expected to decide whether to approve the new drug application in January.
BOSTON (October 7, 2024) – The Barth Syndrome Foundation (BSF), the only patient advocacy organization solely dedicated to Barth syndrome and saving lives around the world through education, advances in treatments and finding a cure, is urging the U.S. Food and Drug Administration (FDA) to approve elamipretide, the first potential therapy for Barth syndrome. The agency’s Cardiovascular and Renal Drugs Advisory Committee is scheduled to review and provide feedback to the FDA related to the New Drug Application (NDA) for elamipretide on October 10.
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...
