The aim is to identify the genetic causes, understand the pathology, prevent the transmission and, finally, develop new therapeutic approaches for these devastating often fatal disorders. (gcb.mumc.nl)
In the long term, however, these findings could contribute to the development of new therapeutic approaches. (sciencedaily.com)
This discovery makes it possible to screen for the disease, and opens the door for potential new therapeutic approaches. (broadinstitute.org)