Rare Diseases

Rare Diseases Rapid Fire Update

Sunday, July 18, 2021

10:05 AM – 10:35 AM ET

Presenter(s)

Nina F. Schor, MD, PhD

Deputy Director
National Institute of Neurological Disorders and Stroke, National Institutes of Health
Bethesda, Maryland, United States
Marc C. Patterson, MD

Professor of Neurology, Pediatrics and Medical Genetics
Mayo Clinic Children's Center
Mayo Clinic College of Medicine and Science
Rochester, Minnesota, United States

Participants should be aware of the following financial/non-financial relationships:

Nina F. Schor, MD, PhD: Disclosure information not submitted.

Marc C. Patterson, MD: Disclosure information not submitted.

Part 1
URGenT NETwork for Ultra-Rare Gene-based Therapy Network – Dr. Schor will explore this new resource from NINDS, which is aimed at enabling providers and institutions with every level of resources and capabilities to make gene therapies available to their patients and families. This segment will outline how the Network will provide consultative services, IND-enabling studies, manufacturing and scale-up of gene and viral constructs, and more, enabling clinicians to support patients and families dealing with rare genetic disorders.