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Gene Therapy
Please join BSF on Thursday, October 15th at 12 pm EDT for a gene therapy webinar with Barry Byrne MD, PhD and Christy Pacak PhD from the University of Florida Powell Center for Rare Disease Research and Gene Therapy, along with Todd Cade PT, PhD from Duke University School of Medicine.

Gene therapy targeting Barth syndrome holds great promise for
our community yet has potential limitations and presents
significant scientific, financial, manufacturing and regulatory
challenges that first must be overcome.

Join this important discussion to:

● Understand the basic concepts as well as some of the complexities
of gene therapy, and how it could be applied as a potential
treatment for Barth syndrome.

● Be informed on the last 24 months of progress in the field of gene
therapy, particularly as a specific application for treating Barth
syndrome within the Powell Center.

● Explore the unique challenges related to the application of gene
therapy in an ultra-rare and multi-system disease such as Barth
syndrome.

Advocacy begins with information. Please plan to join this webinar to become better informed about the potential for gene therapy as a therapeutic option for Barth syndrome in the future. All are welcome; advance registration is required, and this call will be recorded.

For background reading, we recommend:
Piecing together the puzzle: Planning a gene therapy program in Barth syndrome
https://edition.pagesuite-professional.co.uk/html5/reader/production/default.aspx?pubname=&edid=5cf08b2f-f3d9-435a-8255-ef07542eb9a3&pnum=52

Oct 15, 2020 12:00 PM in Eastern Time (US and Canada)

Webinar is over, you cannot register now. If you have any questions, please contact Webinar host: Brett Smith.