What's New

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.

The following is a summary of a live presentation offered to the Barth syndrome patient and family community on March 21, 2018. Stealth BioTherapeutics’ CEO Reenie McCarthy and Chief Clinical Development Officer Jim Carr, Pharm.D., presented an update on Stealth’s clinical trials in mitochondrial disorders and Barth syndrome and answered questions from the community.

This summary and the content within is provided for reference purposes and for the intended audience only. Such reproductions and copies are authorized only when provided directly to the intended audience recipient by Stealth. This summary and the content within does not intend to provide or substitute for medical advice. Please seek the advice of your physician about treatments which may be appropriate for you or your family member.

Volume 18, Issue 1 of the Barth Syndrome Journal is now available online for download.


Powered by Firespring.org