The annual event — held this year on Feb. 28, 2020 — focused on forging connections in the rare diseases community, shortening the journey toward diagnosis and pursuing personalized medicine as a therapeutic approach for treating rare diseases.
NCATS-supported researchers have developed an automated machine-learning approach to diagnose genetic diseases in seriously ill children. This new approach could allow faster diagnosis, initiation of treatment, and, ultimately, better outcomes.
NCATS-supported researchers sent miniaturized tissue chip systems to the International Space Station on May 4. Four projects using the systems will support the study of aging-related conditions and potential treatments for them.