Barth Syndrome Foundation urgently appeals to FDA and Stealth BioTherapeutics to provide access to potential therapy
BOSTON (PRWEB) November 18, 2020
Barth Syndrome Foundation (BSF) today announced a series of advocacy initiatives directed toward U.S. Food and Drug Administration (FDA) and Stealth BioTherapeutics requesting immediate access to the investigational drug elamipretide. Specifically, BSF and the Barth syndrome community are petitioning the drug developer to submit a new drug application (NDA) to FDA requesting approval of the investigational compound elamipretide in people with the ultra-rare and life-threatening disease Barth syndrome. Read more…
BOSTON (PRWEB) AUGUST 24, 2020
Barth Syndrome Foundation (BSF) and American Heart Association (AHA) have announced a first-ever partnership to advance research studying cardiac complications associated with Barth syndrome, a rare, life-threatening, mitochondrial disease. Structured as a fellowship, the partnership aims to also address a severe shortage of early-career researchers by incentivizing a focus on a rare indication. This important collaboration between the AHA and BSF marks a strategic shift by BSF to broaden its impact by joining forces to accelerate progress through science and education. Read more...
Stealth BioTherapeutics Announces Positive Results From Study to Evaluate Efficacy of Elamipretide in Barth Patients Compared to Natural History Control
BOSTON, Feb. 18, 2020 /PRNewswire/ -- Stealth BioTherapeutics (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced positive results from its retrospective natural history study, clinical protocol SPIBA-001, evaluating the efficacy of subcutaneous injections of elamipretide relative to a natural history control comprised of 19 propensity score matched subjects with Barth syndrome (Barth). Subjects receiving elamipretide for up to one year during the TAZPOWER trial and open-label extension showed a mean improvement of greater than 80 meters on their six-minute walk test (6MWT), the primary efficacy endpoint, compared to less than one meter for subjects in the natural history group evaluated over the same time period (p=0.0005). Patients also showed statistically significant improvements in several key secondary endpoints, including muscle strength and sit-to-stand evaluations. Additional analysis of these data will be presented at an upcoming medical meeting.
BOSTON, February 6, 2020 (PRWEB)
Barth Syndrome Foundation (BSF) announced this week Katherine R. McCurdy, MBA (Kate) will assume the role of Chair of the Board of Directors effective April 1, 2020.
McCurdy succeeds Susan McCormack who has led BSF since 2018. During McCormack’s tenure, BSF has made significant strides in translational research and advocacy, most notably hosting an externally-led Patient-Focused Drug Development (PFDD) meeting with the U.S. Food and Drug Administration (FDA) in 2018. In addition, BSF supported the launch of the first two clinical trials in Barth syndrome. “BSF is actively cultivating opportunities to progress from bench to bedside the research that we made possible over the last two decades,” said McCormack. “Kate is uniquely able to lead BSF in this next phase of our work to advance therapies for Barth syndrome.”
BOSTON, Oct. 17, 2019 / PRNewswire
Stealth BioTherapeutics (NASDAQ: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced the presentation of new data from the open-label extension portion of the Phase 2/3 TAZPOWER study evaluating elamipretide in patients with Barth syndrome. The findings, presented at the American Society of Human Genetics (ASHG) 2019 Annual Meeting in Houston, Texas, showed that treatment with elamipretide resulted in a 27% increase in average cardiac stroke volume, or the amount of blood pumped by the heart's left ventricle per contraction, from the trial baseline (40.8 mL) to week 36 (51.8 mL) of the open-label extension. Left-ventricular stroke volume is one of the primary determinants of cardiac output, or the volume of blood pumped by the heart, which is an important indicator of how efficiently the heart can meet the body's demands for perfusion to various organs. In unaffected adolescent boys, an average stroke volume of 85 mL would be expected.
BOSTON (PRWEB) AUGUST 22, 2019
This week, Barth Syndrome Foundation (BSF) announced the appointment of Erik Lontok, PhD, as the Foundation’s new Director of Research. Lontok will succeed BSF’s prior Director of Science, Matthew Toth, PhD, in a new role that has been established in order to leverage the past two decades of research partnerships and achievements toward the development of viable therapies for people with Barth syndrome. Trained as a biochemist at the University of California, San Francisco, Lontok joins BSF after serving as the Chief Science Officer of the Lipedema Foundation.
University Hospitals Bristol NHS Foundation Trust, University of Bristol and Barth Syndrome UK Initiate Phase 2 Study of Bezafibrate in Barth Syndrome
BRISTOL, England (PRWEB) July 24, 2019
University Hospitals Bristol NHS Foundation Trust (UHB), University of Bristol (UoB), Barth Syndrome UK and Barth Syndrome Foundation (BSF) recently announced the opening of CARDIOMAN, a Phase II trial evaluating bezafibrate in individuals affected by Barth syndrome, a rare, life-threatening mitochondrial disease caused by a mutation in the tafazzin (TAZ) gene. The trial, funded by an MRC and NIHR Partnership, is a randomized, placebo-controlled pilot trial to assess the efficacy and safety of bezafibrate to increase mitochondrial biogenesis and potentially modify the cellular ratio of monolysocardiolipin to L4- cardiolipin, a biomarker of Barth syndrome. The trial has been designed in collaboration with the UKCRC-registered Bristol Clinical Trials and Evaluation Unit (part of the Bristol Trials Centre), which has managed the conduct of the study throughout.
BOSTON AND CAMBRIDGE, ENGLAND (PRWEB) FEBRUARY 28, 2019
Healx, a Cambridge (UK) technology company, today announced a collaboration with Boston Children’s Hospital and Barth Syndrome Foundation (BSF) to advance promising therapeutic compounds using a novel approach that will accelerate drug discovery for applications in Barth syndrome, a life-threatening, genetic mitochondrial disease.
Companies such as Healx are capitalizing on advances in artificial intelligence (AI) and merging technology with new findings in science to overcome the financial and time challenges to develop therapies in rare diseases. “We have to consider alternatives to the traditional drug discovery model,” said Dr. Tim Guilliams, CEO and co-founder, Healx. “Rare disease patients simply cannot rely on the traditional drug development process which doesn’t work in a timely fashion for such complex, commonly fatal diseases in niche patient populations.”