On June 19th in Boston, Barth Syndrome Foundation (BSF) conducted a
gathering that included members of BSF’s Board of Directors and the
Scientific and Medical Advisory Board (SMAB), as well as four carefully
selected outside experts,
in order to strategically and tactically evaluate potential therapies
for Barth syndrome nearing clinical development. The review meeting was
intended to identify specific ways BSF can continue to advance the
respective therapeutic ideas which it has helped
to fund over the years through its Research Grant Program, made
possible by the generous support of our donors. This was the first of
what BSF plans will be a continuing series of research review meetings.
Three promising potential new therapies currently in
pre-clinical development by three separate research groups were reviewed in
depth. Notably, BSF supported through its Research Grant Program the
development of each of these potential therapies:
- Gene Therapy (Drs. Barry Byrne, Christina Pacak
and Todd Cade);
- Enzyme Replacement Therapy (Dr. Michael Chin);
- Modifier Gene ALCAT1 (Dr. Roger Shi).
Read greater detail about each of these potential therapeutic areas in the portfolio review summary.
The CARDIOMAN trial, the second clinical trial ever to test a pharmaceutical therapy specifically to treat Barth syndrome, is running a series of successful study days this month in Bristol, UK. CARDIOMAN is evaluating the use of bezafibrate, a drug already approved and used to lower triglycerides, in people with Barth syndrome after a research study in mice demonstrated improvements in heart function and fatigue.
Michaela Damin, Barth Syndrome UK's founder, was inspired by the courage and determination of all the boys and men taking part. "They are incredibly supportive of each other and help each other to find the joy in every moment they get to spend together."
Read the press release about the CARDIOMAN clinical trial -->
By giving to BSF, you will help improve lives through family support programs and propel potential therapies ahead.
Please help us TODAY, so next year we can:
Expand support programs and resources for existing and new families
Attract researchers and clinicians to advance science around gene therapy, enzyme replacement, and the use of existing drugs to treat Barth syndrome
Collaborate with FDA and industry to bring about more clinical trials in Barth syndrome
Improve tools to further a positive experience for families, researchers, and healthcare providers at the 2020 BSF International Conference
In July 2020, BSF will celebrate their 20th anniversary and hold the 10th International Scientific, Medical and Family Conference in Clearwater Beach, Florida. Community and collaboration are the building blocks of this global gathering. In addition, significant research accomplishments have been achieved as a direct result of the collaboration of families alongside clinicians and researchers at this biannual meeting. Read our featured community story about the history of the BSF International Conference.
We just published our 2018 Annual Report, and we hope you will take time to read stories from our community and articles describing global research efforts. Click on the image to read the report fullscreen, or download a PDF here.