Learning From Nano-rare Patients | 2023 Nano-rare Patient Colloquium 

Advances in science are incremental and each additional bit of knowledge contributes to the evolution of understanding. No area of scientific inquiry is more incremental than advances in the treatment of diseases. What can be learned by addressing the needs of patients with the rarest of mutations that cause a more common rare genetic disease is immediately applicable to the entire group of patients with the disease. In this panel, we discuss how studying a patient or a few patients, even if they have the rarest of the mutations known to cause the disease, can expand our knowledge about the molecular causes of a disease and potential value of therapeutic interventions.
Moderator
Andrew Marshall, Ph.D.
Venture Partner, Scion Life Sciences
Panelists
Jeff Carroll, Ph.D.
Associate Professor in the Department of Neurology, University of Washington
Barbara Cheifet, Ph.D.
Chief Editor, Springer Nature Group
Vijay Ganesh, M.D., Ph.D.
Associate Neurologist Instructor, Harvard Medical School
John Spiro, Ph.D.
SVP & Sr Scientist, SFARI