ASGCT Policy Summit Highlights Patient and Advocate Perspectives
ASGCT Staff - October 14, 2019
ASGCT will have patient, caregiver, and advocate speakers participating in all three days of our upcoming Policy Summit, held at The Westin in Washington, D.C. November 4-6.
On the first day of the Policy Summit, attendees will hear from regulators from five countries about enhancing convergence of regulatory requirements internationally. The day will close with a reminder of the importance of expediting the availability of gene therapies.
Jeanette Hogan is the mother of Jack Hogan, who was the first-ever recipient of Luxturna in 2018. Jeanette will address how her son’s treatment changed his life.
Paying for one-time, potentially curative gene and cell therapies under current reimbursement mechanisms can pose challenges. The second day of programming will address payment policy issues and innovative solutions. Two parent caregivers will speak on a panel about the value of gene therapies which, for patients and their families, goes far beyond dollars and cents.
Jenn McNary’s sons Max and Austin have both been diagnosed with Duchenne Muscular Dystrophy (DMD). Jenn will talk about the heavy burden that diseases like DMD have on a family; the physical, social and economic impact that gene therapy may provide for patients; and concerns about patient access to treatment upon approval.
Khrystal Davis, J.D., is the founder and president of the Texas Rare Disease Alliance. Her son Hunter was diagnosed with spinal muscular atrophy (SMA) type 1, the leading genetic cause of mortality in children under the age of two. Khrystal will address the difference gene therapy can make in treating this disease before symptoms progress, and the implications of treatment on the healthcare payment system.
The third day of programming will be devoted to the ethics, societal impacts, and policy issues surrounding the clinical use of germline, or heritable, gene editing. A featured panel of individuals who are directly impacted by the debate will discuss their perspectives on the clinical use of germline gene editing.
Neena Nizar, Ed.D., is the president and founder of The Jansen’s Foundation. She and her two sons, Arshaan and Jahan, are among only about 25 people worldwide to be diagnosed with Jansen’s disease. Neena will talk about the balance of social and bioethical issues from the unique perspective of both a parent and patient.
Andrea Taylor’s son Aiden was diagnosed at age two with arterial tortuosity syndrome. Andrea went on to found and lead A Twist of Fate-ATS, and will talk about her family’s experiences.
Julia Bascom is the executive director of the Autism Self-Advocacy Network. She has spent her career advocating for neurodiversity and disability rights, speaking on the distinction between disease and disability, and the ethical problems of deciding which lives are worth living.
Register for the Policy Summit today
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