Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults
for which there is presently no therapy. Recently, a study reported that interferon gamma
(IFN-g) could raise frataxin protein levels in both cell lines derived from patients with
Friedreich ataxia and in a mouse model with Friedreich ataxia. The present study will test
whether IFN-g is safe, tolerated and potentially efficacious in a heterogeneous cohort of
children with FRDA.
Phase:
Phase 2
Details
Lead Sponsor:
Children's Hospital of Philadelphia
Collaborators:
Friedreich's Ataxia Research Alliance Vidara Therapeutics Research Ltd