The elections will take place from 23 May - 5 June 2022 and is held online via the link https://icnapedia.org/ballot-2022 . All eligible voters will be sent their unique codes during the perior 23 May 2022 - 25 May 2022 . The codes are sent out in batches. Please only contact us if you have not received your unique voting code by 26 MAY 2020. Meanwhile we would strongly encourage you to go through the candidate statements , which are now available online. Any new ICNA members, including those who have been converted to FULL Membership from JUNIOR Membership after 23 May 2020 will NOT be eligible to vote in this election. The election site will be open tilll 5 June 2022 6PM CET.
Participants will gain an understanding of the rapidly evolving landscape of gene therapy for neurological disorders of childhood. We will discuss the basic principles of gene vector development and delivery, and describe specific diseases for which gene therapy is currently approved for clinical use or available as an experimental treatment in clinical trials. Finally, we will discuss challenges facing gene therapy development, including cost and worldwide accessibility.
- Understand the principles of gene therapy development and delivery.
- Be able to describe how different gene therapy strategies can be used to treat specific neurologic diseases in children.
- Recognize genetic disorders that are amenable to treatment with gene therapy.
- Identify opportunities for patients with rare diseases to participate in clinical trials of experimental therapies.
Toni Pearson, MBBS
Washington University School of Medicine, St. Louis, Missouri, USA
Gene Therapy Primer
Barry Byrne, MD, PhD
Child Health Research Institute, University of Florida, Gainesville, Florida, USA
Experiences with Gene Therapy for Childhood Neuromuscular Disorders
Diana Bharucha-Goebel, MD
Children’s National Hospital & National Institutes of Health, Washington, DC, USA
Targeting the Central Nervous System: Experiences with Gene Therapy for AADC Deficiency
Toni Pearson, MBBS
Panel Discussion – Present and Future Challenges in the Development of Universally Accessible New Therapies for Ultra-Rare Diseases
Carsten G. Bönnemann, MD
National Institute of Neurological Disorders and Stroke, Bethesda, Maryland, USA