Nina Schor, MD, PhD, FAAN, National Institute of Neurological Disorders and Stroke, Bethesda, MD, shares her thoughts on the difficulties in treating ultra-rare neurological diseases and the subsequent need for the Ultra-rare Gene-based Therapy (URGenT) network to tackle this. Dr Schor explains that it is difficult to develop therapies for such unique disorders due to discrepancies in mutations resulting in patients being unable to utilize generalized molecular therapies. Additionally, there is a lack of commercial interest in developing therapies for these rare disorders due to complex financial and geographical factors affecting the therapeutic market. The URGenT network aims to increase the availability of therapies for these rare diseases, benefitting patients and physicians who will be able to access the necessary technology and resources for treatment. This interview took place at the American Academy of Neurology 2022 Congress in Seattle, WA.
