With a strong foundation of innovative research and multidisciplinary collaboration, the Center for Rare Neurological Diseases seeks to develop new treatments for devastating neurological diseases that affect children and adults.
While the number of patients who suffer from neurological conditions such as Alzheimer's disease and Parkinson's disease is on the rise, developing treatments for these disorders has proven difficult. Recent changes in healthcare and diminishing research budgets have also posed a challenge for academic institutions pursuing biomedical research. We believe that these challenges are also opportunities to learn from past experiences and establish new directions in translational neurosciences.
The Center’s research approach is based on the premise that rare genetic diseases offer a unique opportunity to develop targeted therapies for neurodegenerative disorders across the lifespan. We are focusing on lysosomal disorders that are clinically and genetically linked to more common conditions such as Parkinson’s and Alzheimer’s in order to identify novel targets for therapeutic development. By pursuing these targeted therapies – through the collaborative efforts of Northwestern University experts in nanotechnology, biomedical engineering, chemistry, and genomics – we hope to transform and personalize care for patients with neurodegenerative diseases.
Rare diseases are also referred to as neglected or orphan diseases, since they affect underrepresented populations that seldom attract enough research support or funding to pursue the development of therapies. We are passionate about identifying disease mechanisms and targets that are shared between rare and common disorders in order to concurrently develop treatments for both types of conditions.
It is an exciting time for neurology and by working together we have the potential to find treatments for many incurable neurological disorders. We owe this to those who suffer from these disorders and their families.
We welcome your feedback. Please contact us with you questions and comments.
Dimitri Krainc, MD, PhD
Director, Center for Rare Neurological Diseases
Aaron Montgomery Ward Professor and Chair
Ken and Ruth Davee Department of Neurology