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Gene Therapy in Barth Syndrome: A Community Roundtable

Gene therapy holds immense promise for our community, and we invited Drs. Barry Byrne, Christy Pacak, and Todd Cade, to discuss the challenges facing the entire gene therapy field, from immune management to regulatory issues and trial design.


Webinar Recordings (2021)

Webinar Recordings (2020)

Recordings and Written Summaries

(2021)

(2020)

Webinar Recordings (2019)

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