Friedreich ataxia: from genes to therapies?
Most cases are caused by a single mutation, paving the way for therapeutic advances for this fatal disease.
Delatycki
Martin B
MB
Ioannou
Panos A
PA
Churchyard
Andrew J
AJ
D005621:Friedreich Ataxia / Q000188:drug therapy* / Q000235:genetics*; D006801:Humans; D012106:Research