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Gene Therapy in Barth Syndrome Town Hall

Thursday, March 2, 2023
12:00 pm1:00 pm

We have heard and learned over the years about the immense potential of gene therapy for Barth syndrome - where delivery of a new copy of the gene, TAFAZZIN, is meant to impact the cause of this disease. Please join us for a conversation about current efforts in this field, including the Bespoke Gene Therapy Consortium, for which Barth syndrome was one of 14 fields invited to apply. We welcome your questions about how gene therapy works, what it potentially means for you or your loved ones, and who is working to maximize safety and measure effectiveness of the potential therapy.

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