URBC now in Center of Excellence network for Batten disease
Program seeks to improve outcomes, help patients obtain top care
The University of Rochester Batten Center (URBC) was designated a Center of Excellence by the Batten Disease Support, Research, and Advocacy (BDSRA) Foundation in recognition of its efforts at advancing care and research for Batten disease.
The BDSRA Center of Excellence Program seeks to improve outcomes in Batten disease and improve the quality of life of those with the condition by fostering access to the best clinical care via its U.S. network of centers, along with investing in research toward new treatments.
URBC, which is part of the University of Rochester Golisano Intellectual and Developmental Disabilities Institute, is one of the five institutions named as Centers of Excellence. Others include Children’s Hospital Colorado, Children’s Hospital of Orange County in California, Kennedy Krieger Institute in Baltimore, and Nationwide Children’s Hospital in Ohio.
“This recognition underscores our commitment to providing comprehensive care to patients and their families while advancing critical research,” Jennifer Vermilion, MD, a pediatric neurologist and the director of URBC, said in a university press release. “With this support, we will continue our efforts to develop new therapies and improve the quality of life for those affected by Batten diseases.”
A new Center of Excellence
Batten disease, also called neuronal ceroid lipofuscinosis (NCL), belongs to a group of inherited neurological conditions marked by the toxic accumualtion of lipofuscins, substances composed of fats and proteins.
Nerve cells are particularly sensitive to this accumulation, which leads to symptoms such as a decline in motor and cognitive capabilities, along with vision loss and seizures. The disorder is classified into five different types — congenital, infantile, late infantile, juvenile, and adult Batten disease — based on age and symptom severity.
The URBC adopts a comprehensive, multidisciplinary strategy for treating Batten disease, ensuring patients benefit from a coordinated and collaborative care plan delivered by a diverse group of experts that includes neurologists, geneticists, ophthalmologists, neuropsychologists, and other healthcare professionals.
The new Center of Excellence has several research projects aimed at shedding light on the mechanisms underlying Batten disease to develop more effective therapies. Projects are focused on describing the natural history of the distinct forms of the disease, as well in identifying biomarkers for early diagnosis. Tackling the genetic causes of Batten and developing potential gene therapies are other research avenues being pursued at URBC, along with clinical trials.
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