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)

• Barth Fatigue, presented by Stacey Reynolds, PhD, 18-June-2021
• COVID-19 Vaccine Antibody Response in Transplant Patients, presented by Brian Boyarsky, MD, PhD, 27-May-2021 Written Summary

Webinar Recordings (2020)

• Gene Therapy, presented by Barry Byrne, MD, PhD, Christy Pacak, PhD, W. Todd Cade, Pt, PhD, 15-Oct-2020
• Assessment of Cardiac Function in Barth syndrome, presented by Dr. Carolyn Taylor, Medical University of South Carolina, 19-Aug-2020
• 2020 Scientific and Medical Symposium Presentations and Abstracts, 23-24-Jul-2020 
• Cardiac Aspects of Barth Syndrome: Electrophysiology, presented by Dr. Lanier Jackson and Dr. Carolyn Taylor, Medical University of South Carolina, 16-Jun-2020 
• Potential Role of Entresto (sacubitril-valsartan) in Barth Syndrome Cardiac Management, presented by Dr. John Jefferies, University of Tennessee Health Sciences Center, 19-Feb-2020 

Recordings and Written Summaries

(2021)

• A Conversation With Todd Cade, September 24, 2021
• Community Check-in, August 27, 2021
• Project Sunshine, with Erika Apupalo and Kyra Hu, 23-July-2021
• Extraordinary! A book for children with rare diseases, with Evren and Kara Ayik 25-June-2021
• COVID-19 Vaccine Experience, with Terry Jo Vetters, 8-Jan-2021

(2020)

• Ask the Doctor, with Richard Kelley, MD, PhD Medical Genetics, 13-Nov-2020
• How to Advocate for Yourself in an Ableist World, with Brett Smith, 6-Nov-2020
• Recognizing Fatigue and Conserving Energy in Barth Syndrome, with Cristy Balcells, RN, MSN 30-Oct-2020
• The Perspective of Men with Barth Syndrome: Relationship Building, Family Planning, and Reproductive Decision-Making, with Jason Shandler, Boston University School of Medicine, 23-Oct-2020
• Metabolic Management of Barth Syndrome, with Richard Kelley, MD, PhD, Medical Genetics, 9-Oct-2020
• Life Hacks:  Navigating Social Security with Brett Smith, 2-Oct-2020
• Advocacy in Action TAZPOWER Clinical Trial with Hilary Vernon, MD, TAZPOWER Clinical Trial PI, 25-Sep-2020
• Advocacy in Action with Walker Burger TAZPOWER clinical trial participant and James Valentine BSF Regulatory Counsel, 18-Sep-2020
• Barth Syndrome Community Voice Report with Maria Picone, CEO, Founder TREND Community, 28-Aug-2020
• Barth Syndrome Carrier Related Issues, with Rebecca McClellan, MGC, CGC, Genetic Counselor, Center for Inherited Heart Disease, Johns Hopkins Medicine, 21-Aug-2020
• Primer in Barth Syndrome Metabolism, with Richard Kelley, MD, PhD, Medical Genetics, 14-Aug-2020
• Etiology of Eating Orality Disorder in Barth Syndrome, with Florence Mannes, Speech Therapist, Medicine Sorbonne University, 7-Aug-2020
• Back to School Beyond the Backpack: COVID-19 Concerns when weighing the decision about returning to school with B.J. Develle, LCSW 31-Jul-2020
• Gene Replacement Therapy in the Barth syndrome Knockout Mouse with Dr. Suya Wang and Dr. Bill Pu from Boston Children's Hospital, Harvard Medical School, 26-Jun-2020, Written Summary
• Strength, stamina and fatigue in Barth syndrome, with  Brittany DeCroes Hornby, PT, DPT, PCS from the Department of Physical Therapy, Kennedy Krieger Institute, 5-Jun-2020,  Written Summary

Webinar Recordings (2019)

• Barth Syndrome Foundation 2019 Town Hall Meeting, highlighting our success to further the mission of BSF in education, advances in research, and finding a cure, 12-Jun-2019