The Barth Syndrome Registry and Repository (BRR) stores information about individuals affected with Barth syndrome. Affected individuals or their family members can register for the BRR, consent to participate, and provide the following information on the affected individual: medical records, genetic test results, biological samples, demographic information, and responses to a battery of survey assessments (clinical information, quality of life, medical procedures, and family history).
The goal of the BRR is to build the natural history data on the disease and further the understanding of Barth syndrome.
To learn more about the Barth Syndrome Registry go to www.barthsyndromeregistry.org.
BSF 9th International Scientific, Medical & Family Conference
July 20, 2018 ~ Clearwater Beach, FL
Summarized findings from the Barth registry interviews: Key points for moving forward
Anthony Aiudi, PharmD, Stealth Biotherapeutics, Newton, MA