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Goals and Mission

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.