What's New

The Barth Syndrome Foundation, Inc. (BSF) and its international affiliates announce the availability of funding for basic science and clinical research on the natural history, biochemical basis, and treatment of Barth syndrome. There are two basic categories: IDEA grants for 1-2 years and DEVELOPMENT grants for 2-3 years with budgetary maximums of US $50,000 or $100,000, respectively. Although BSF will consider any research proposal related to Barth syndrome, it is particularly interested in supporting research in the areas identified by REQUEST FOR APPLICATIONS (RFAs) that are posted on its website. RFAs for work in clinical/scientific areas that BSF considers to be high priority areas of investigation may have increased budgetary maximums and other requirements (see the BSF website for details about any RFAs). Applications responding to RFAs will be given preferential consideration in the BSF Research Grant Program.

BSF's Research Grant Program now requires all applicants to be independent investigators (e.g., faculty appointment). Postdoctoral fellows cannot apply. BSF allows young, non-tenured investigators to include in their submitted budget up to 75% of the total grant amount as PI salary. In addition, for those clinical applications where volunteers must travel to a clinical research site, these travel expenses will be handled separately and will be excluded from the budget maximums mentioned above. We encourage independent investigators at all professional levels to submit their best ideas. There are no geographical limitations to this funding.

Deadline

The deadline for submission of the completed research grant application is October 31, 2018, and grants will be awarded in late February, 2019. The deadline for the one-page "Letter of Intent," if applicable, is September 21, 2018.

William Blair, an investment bank and asset management firm based in Chicago, ran a short feature about Barth Syndrome Foundation (BSF) in their quarterly newsletter, Client Focus. The recent issue was recently released and highlights BSF connecting affected individuals around the world and their families to create a virtual community and supportive environment of those living with Barth syndrome. It’s a beautiful testament about the power of community, near and far, made possible through social media.

Alignment around a cause is the most enduring and powerful level of alignment. As Steve McCurdy was telling me about the most recent Barth Syndrome Foundation (BSF) conference, the power of a compelling cause to make people put aside any petty jealousies and conflicts was clear. This permeates everything about BSF from its strategy and culture to organization and ecosystem and what they do. Learn more...

Multi-scale Modeling of Inherited Pediatric Cardiomyopathies

William Pu, MD, a Scientific and Medical Advisory Board member of BSF and Kit Parker, PhD are Principal Faculty members at the Harvard Stem Cell Institute (HSCI), and they have joined together to make laboratory heart models more physiologically relevant to patients with heart disease. Barth syndrome is one of the three heart disease models chosen to be developed. Drs. Parker and Pu are collaborating using a prestigious Research Project Cooperative Agreement from the NIH (“Multi-scale modeling of inherited pediatric cardiomyopathies”—UG3HL141798) to make a 3-dimensional actively-beating model of the heart ventricle using induced pluripotent stem cells as starting material. This work continues the “heart on a chip” model that both researchers published upon in 2014. Congratulations to both Drs. Parker and Pu for receiving this prestigious NIH award! Their proof-of-concept study of the technology has just been published in Nature Biomedical Engineering, and disease models, including Barth syndrome, based on patient cells are in the works.

https://hsci.harvard.edu/news/heart-research-gets-better-3d-model

https://www.nature.com/articles/s41551-018-0271-5

Are you participating in the TAZPOWER trial using Barth syndrome treatment injections developed by Stealth BioTherapeutics? If so, you and your caregiver or someone close to you are invited to participate in another study sponsored by Stealth.

This is a quick, in-home study that gives people with Barth syndrome and their caregivers/observers the opportunity to explain any ways in which patient functioning and lifestyle may have changed during the TAZPOWER trial in their own words. Patients and their caregivers/observers will simply download an app on their phone and use that app to video record an interview about the way patients felt and functioned during the trial. The answers provided will be combined with the data captured during the trial to develop a full picture of the treatment’s impact on the patients’ lives.


Powered by Firespring