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Research Summaries

Research Summaries for the Barth syndrome Community

Click below to view summaries of recent research in Barth syndrome

Gene therapy and the role of exercise

How loss of TAFAZZIN function ignites mitochondrial dysfunction

Cell stress response impacts energy use in the heart

Evaluation of Barth syndrome manifestations in female carrier mice

Impact of raising children with Barth syndrome on parental health-related quality of life and family functioning

What is the Difference Between Preprint vs Peer Review

Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER

The diagnostic odyssey, clinical burden, and natural history of Barth syndrome: an analysis of patient registry data 

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