"There is a great sense of community and interconnectedness among the folks who are here," said an attendee of the 10th Annual Friedreich's Ataxia Symposium.
A new approach being developed at The Children’s Hospital of Philadelphia and the University of Pennsylvania could allow investigators to know within days if a chosen therapy reverses the intracellular mechanisms that go awry in Friedreich’s Ataxia (FA), a genetic mitochondrial disease
The Wilson Lab studies the inherited neuro- and cardio-degenerative mitochondrial disorder, Friedreich ataxia (FA). The lab also constructed the first random shRNA-expressing libraries and is using these libraries for drug discovery in a variety of disorders.
