Grant number: U54 HD122210-01

Principal Investigator: Erika Augustine, MD, MS

Lead Institution: Kennedy Krieger Institute

Diseases Studied

Batten Disease is a group of rare, inherited nervous system disorders. It is caused by genetic changes that disrupt the body’s ability to remove cellular waste, leading to a buildup of harmful materials in brain and nerve cells. Over time, this causes progressive neurological decline, including vision loss, seizures, cognitive impairment, motor difficulties, and often shortened lifespan.

Batten Disease is also called neuronal ceroid lipofuscinoses (NCLs). There are 13 recognized NCLs, which vary by age of onset, symptoms, and rate of progression depending on the specific gene involved.

Funding Opportunity

The mission of the Batten Disease Clinical Research Consortium (BDCRC) is to establish and maintain a multi-site clinical and translational research program that drives rigorous and reproducible science, high data quality, operational efficiency, and strong community partnership, in service of creating novel neuronal ceroid lipofuscinosis (NCL) therapies, continuously improving care standards, and advancing knowledge. The Early Career Investigator Grant (ECIG) addresses training needs in rare disease research by providing highly specialized education in the complexities of clinical research in the NCLs. We expect the ECIG will accelerate the careers of emerging investigators who have demonstrated exceptional potential to become independent clinician-scientists.

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Funding Statement

The Batten Disease Clinical Research Consortium (BDCRC) is part of the Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH) and led by the National Center for Advancing Translational Sciences (NCATS) through its Division of Rare Diseases Research Innovation (DRDRI). BDCRC is funded under grant number U54HD122210 as a collaboration between NCATS, the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), and the National Institute of Neurological Disorders and Stroke (NINDS). This website is hosted by the network’s Data Management and Coordinating Center at Cincinnati Children’s Hospital Medical Center, which is funded by NCATS and NINDS under grant number TR002818.