Accelerated Investment Propels Efforts to Revolutionize Therapeutic Approaches for Uncommon Nervous System Ailments
University of Rochester Medical Center Leads the Way in Gene Therapy Trials for Rare Neurological Diseases
The University of Rochester Medical Center (URMC) is at the forefront of revolutionizing the treatment of rare neurological diseases, thanks to its significant role in gene therapy clinical trials.
URMC is spearheading groundbreaking research in this field, with the initiation and conduct of first-of-their-kind gene therapy studies. One such study focuses on Batten disease, a rare neurological disorder, demonstrating URMC's leadership in developing innovative therapeutic interventions for these conditions.
The University's participation in the National Institute of Neurological Disorders and Stroke (NINDS) Network for Excellence in Neuroscience Clinical Trials has further bolstered its capacity. This network provides URMC with access to advanced neuroscience research infrastructure and collaboration opportunities, essential for designing, implementing, and assessing sophisticated therapeutic protocols in rare neurological conditions.
The collaboration enables URMC to contribute to advancing the clinical trial landscape by enabling rigorous evaluation of gene therapies' safety and efficacy, accelerating the translation from research to patient care.
A team of dedicated professionals, including Emma Ciafaloni, MD, Jennifer Vermillion, MD, Charles White, MD, and Robert Holloway, MD, MPH (who serves as the principal investigator of the Rochester site, named UR-NEXT), and Christine Annis (program coordinator for UR-NEXT), are driving this research forward.
The new funding brings the medical center's total federal funding for the program to $5.7 million, further enhancing their ability to overcome recruitment and retention barriers associated with rare diseases and measure the impact of treatments based on outcomes that are important to patients and their families.
As the field of neurology undergoes a transformation with the development of new gene therapies for a wide range of rare and common neurological and neuropsychiatric diseases, URMC's role as a key site for advancing gene therapy trials targeting rare neurologic diseases is more crucial than ever. This collaboration facilitates the development of innovative treatments and improves patient access to experimental therapies under rigorous scientific oversight.
- The University of Rochester Medical Center (URMC) is leading the science of gene Therapy trials for various medical-conditions, particularly rare neurological disorders, by initiating and conducting unique gene therapy studies.
- URMC's collaboration with the National Institute of Neurological Disorders and Stroke (NINDS) Network for Excellence in Neuroscience Clinical Trials has bolstered their capacity, providing access to innovative science research infrastructure and fostering collaboration that aids in designing, implementing, and assessing advanced therapies and treatments for neurological disorders.
- The clinical trials conducted by URMC are crucial for neurological disorders, as they facilitate the development of innovative therapies and treatments, improve patient access to experimental therapies under rigorous scientific oversight, and measure the impact of treatments based on outcomes that are important to patients and their families.
