The main objective of the Scientific and Medical Conference about Barth Syndrome is translation of scientific advances discussed at this Conference into therapy for rare diseases like BTHS. The strategy is to assemble interested physicians/researchers to communicate experimental results and clinical findings and stimulate new work, collaborations, and publications. This Conference also provides a forum for critically discussing clinical advances or potential treatments.
Dr. Charlene Schramm,
National Heart, Lung, and Blood Institute
Office of Rare Diseases Research