Monday, August 11, 2014
AAVLife is developing an adeno-associated viral
vector-based gene therapy that could become the first
disease-modifying treatment for Friedreich's ataxia-associated cardiomyopathy.
The therapy has the potential to decrease the risk of cardiac events and
improve patient survival.
Friedreich's ataxia is a rare hereditary progressive neurodegenerative
condition caused by mutations in the gene encoding frataxin (FXN; FRDA) that
result in decreased expression of the mitochondrial protein.