Barth Syndrome Foundation

On September 6th, the U.S. Food & Drug Administration (FDA) announced that the Cardiovascular and Renal Drugs Advisory Committee has scheduled their meeting to review Stealth Biotherapeutic Inc.'s new drug application (NDA) 215244 for elamipretide hydrochloride injection for the treatment of Barth syndrome for October 10th, 2024. The global Barth syndrome community has a valuable role to play in the FDA’s review of elamipretide hydrochloride. As part of their review, Advisory Committee members will have an opportunity to read letters submitted from the Barth syndrome community, so they better understand the experience of living with and caring for someone with Barth syndrome. This written comment opportunity is open to all members of the Barth syndrome community worldwide, not just US residents, but all letters must be submitted in English. Letters must be submitted no later than September 26. Contact Shelley Bowen: shelley.bowen@barthsyndrome.org to learn more and get instructions on how to submit your letter.

The 2025 grant cycle is now open, and Barth Syndrome Foundation (BSF) and our International Affiliates welcome innovative applications that address the basic, translational, and clinical research challenges of Barth syndrome. Barth Syndrome Foundation seeks to provide seed grant funding to young and established investigators to help catalyze generation of preliminary data that are required for successful follow-on funding from institutions like the National Institutes of Health. Please feel free to share with the physicians and researchers in your life! Applicants must be independent investigators (e.g. faculty appointment). Pre- and postdoctoral applicants are encouraged to apply to the American Heart Association & Barth Syndrome Foundation Predoctoral and Postdoctoral Fellowship program (Deadline: 9/4/24).   Application Details: Up to $100,000 USD maximum total cost, 1-3 years in duration. The number of grants to be funded is dependent on funds available each year. Full Applications are due Friday, November 1, 2024. To learn more about Barth Syndrome Foundation's 2024 grant program, including full guidelines and to apply, click below. https://lnkd.in/dQXsHCz5

What a wonderful time the Stealth team had connecting and re-connecting with #BarthSyndrome advocates, innovators, and affected individuals at the 2024 Barth Syndrome Foundation Conference! This #RareDisease community is a special group of people – with tremendous unmet medical needs. We remain committed all those affected by this life-threatening cardiac disease.

🌟 Today, we experienced heartwarming moments with the Barth Syndrome Foundation, alongside families, medical professionals, researchers, and supporters. Our Chief Scientific Officer Dr. Yingbin Ouyang had a fantastic day, and we're dedicated to supporting rare disease research and families.💜💛💚 📌 At Cyagen, we're your committed partner in accelerating rare disease research. Beyond our HUGO-GT program (https://lnkd.in/gBe5BuPy), we provide a FREE data platform offering comprehensive resources on rare disease epidemiology, drugs, genetics, mutations, and animal models (https://lnkd.in/gF7ZFbe7). Together, we can make a difference!🙌 #RareDiseaseResearch #BarthSyndrome #Cyagen #HUGOGT #MedicalResearch #SupportFamilies #researchmatters #researchimpact #sciencecommunication #scienceandtechnology #biotech #biotechnology

Amazing week at Barth Syndrome Foundation International Scientific Medical and Family Conference. Inspiring to be with a patient community so deeply dedicated to progressing scientific research to improve patient care.

🌟 Today, we experienced heartwarming moments with the Barth Syndrome Foundation, alongside families, medical professionals, researchers, and supporters. Our Chief Scientific Officer Dr. Yingbin Ouyang had a fantastic day, and we're dedicated to supporting rare disease research and families.💜💛💚 📌 At Cyagen, we're your committed partner in accelerating rare disease research. Beyond our HUGO-GT program (https://lnkd.in/gBe5BuPy), we provide a FREE data platform offering comprehensive resources on rare disease epidemiology, drugs, genetics, mutations, and animal models (https://lnkd.in/gF7ZFbe7). Together, we can make a difference!🙌 #RareDiseaseResearch #BarthSyndrome #Cyagen #HUGOGT #MedicalResearch #SupportFamilies #researchmatters #researchimpact #sciencecommunication #scienceandtechnology #biotech #biotechnology

Are you attending the Barth Syndrome Foundation Conference 2024, the Biennial International Scientific, Medical, and Family Conference? Don't miss the opportunity to talk with our esteemed Chief Scientific Officer, Dr. Yingbin Ouyang. Together, we can support Barth syndrome research and make significant contributions to rare diseases.❤️💙💚💛💜 Cyagen's HUGO-GT™ program offers advanced humanized mouse models for better studying human diseases and drug development. Utilizing TurboKnockout-Pro technology, these models replace mouse genes with full-length human sequences, overcoming the limitations of traditional transgenic models. They enable precise gene expression and regulation, closely mirroring human biology. ➡ Learn more here: https://lnkd.in/gBe5BuPy #BarthSyndrome #RareDiseaseResearch #ScientificConference #MedicalResearch #GeneTherapy #HumanizedMice #DrugDevelopment #ResearchInnovation #Cyagen #biotech #biotechnology #scienceandtechnology #researchmatters

The 2024 Barth Syndrome International Conference begins TODAY at the beautiful Hyatt Regency Coconut Point Resort and Spa in Bonita Springs, FL! We are thrilled to welcome our diverse network of healthcare providers, researchers, caregivers, and affected individuals for a week of learning, sharing, and togetherness. This year, we are proud to offer Continuing Medical Education (CME) credits for the first time, furthering our commitment to advancing knowledge and care in the Barth Syndrome community. We look forward to an inspiring week filled with impactful sessions, meaningful connections, and the collective drive to make a difference. 

We’re looking forward to seeing so many in the #BarthSyndrome community at the Barth Syndrome Foundation International Conference, starting this weekend! We’re continuing our commitment to advancing knowledge of this #RareDisease and sharing data on our investigational therapy. Check out the presentations by James Carr, our Chief Clinical Development Officer, and the three posters authored by Hilary Vernon of the The Johns Hopkins University School of Medicine.  Conference Website: https://bit.ly/3WxWYXh

🚀 Exciting News! We will be at #BSF2024 with Barth Syndrome Foundation! 🎉 Visit us to meet our experts and discuss the latest advancements in rare disease research. Explore collaboration opportunities and discover how we can support your research and development goals. 📅 Date: July 29 - August 3, 2024 📍 Venue: Bonita Springs, Florida Don’t miss this chance to engage with us! 🤝 Learn more about how our HUGO-GT can assist your rare disease research here: https://lnkd.in/gBe5BuPy We look forward to seeing you there! #barthsyndrome #rarediseases #rarediseasesresearch #preclinical #preclinicalresearch #scienceandtechnology #biotech #biotechnology #biosciences #eventnews #researchimpact #researchmatters