Led by Dr. Michael Benatar and Joanne Wuu, the ALS Research Collaboration (ARC) at the University of Miami represents a coordinated effort to understand the reasons why we lack effective therapies for patients with ALS, and to advance scientific progress towards a meaningful treatment for this devastating disorder. The many challenges to therapy development include the diversity of factors that may cause the disease, the complex and incompletely understood array of biological mechanisms that mediate disease, the limitations of existing pre-clinical models of disease, the varied clinical phenotype, the paucity of validated biomarkers, and the likelihood that the disease process begins well before symptoms first appear.
It is our mission to overcome these challenges and to help fill gaps in our knowledge by studying genetic and environmental risk factors for disease, illuminating the natural history of disease specifically as it pertains to different genetic causes of disease, probing the relationship between genotype and phenotype, aiding in the development of potential biomarkers and utilizing these to study pre-symptomatic disease, and evaluating new therapies through clinical trials.
Our work is predicated upon the principles of collaboration and partnership, and we work collaboratively with researchers across the United States, Europe and Australia. Moreover, our work is motivated by the needs of our patients and in turn can only succeed through a true partnership with our patient community and their families.