Batten Disease Clinical Research Consortium

The Batten Disease Clinical Research Consortium (BDCRC) is part of the [Rare Diseases Clinical Research Network](https://rarediseasesnetwork.org/) (RDCRN), which is funded by the National Institutes of Health (NIH) and led by the [National Center for Advancing Translational Sciences](https://ncats.nih.gov/ "Learn more about NCATS. Opens in New Window.") (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](https://www.nichd.nih.gov/ "Learn more about the NICHD. Opens in New Window.") (NICHD), and the [National Institute of Neurological Disorders and Stroke](https://www.ninds.nih.gov/ "Learn more about NINDS. Opens in New Window.") (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. The content of this website is solely the responsibility of the BDCRC administrative coordinating center at Kennedy Krieger Institute and does not necessarily represent the official views of the NIH. **Our Mission** We work together to advance patient-centered research and speed the development of effective, accessible treatments for everyone affected by NCL. **Goals** 1. Provide administrative structure to orchestrate and facilitate the efforts of the BDCRC Cores, Projects, and external collaborators, in collaboration with the RDCRN. 2. Meaningfully partner with the broader NCL community in all BDCRC activities to elevate stakeholder priorities and increase the impact of our work. 3. Integrate with, contribute to, and access the robust resources of the DMCC and broader RDCRN to ensure an optimal and efficient BDCRC program. 4. Train, mentor, and inspire the next generation of NCL clinician-scientists (Career Enhancement Core). 5. Comprehensively support rigorous pilot studies with potential to scale to future BDCRC projects (Pilot Study Core). 6. Characterize the natural history of NCL diseases, develop and identify optimal tools for symptom and disease measurement, and test methods that will advance NCL knowledge and enable rigorous clinical trial design in the future (Research Projects). 7. Develop enduring resources for future clinical and translational research.