The investigational therapy DT-216 safely worked to more than double the activity of the FXN gene — involved in energy…
Articles by Steve Bryson, PhD
Repeat use of G-CSF stem cell therapy, an approved medicine, was safe in adults with Friedreich’s ataxia and associated with…
Levels of the protein frataxin, which are abnormally low in people with Friedreich’s ataxia (FA), are further reduced by…
Children and adolescents with Friedreich’s ataxia (FA) who have an enlarged heart were found to have a high prevalence…
Increased sodium levels were found in regions of the brain commonly affected by Friedreich ataxia (FA) — the cerebellum…
Compounds that blocked the activity of histone deacetylases — enzymes that suppress gene activity — increased the activity of the…
Reata Pharmaceuticals has completed a rolling submission of a new drug application (NDA) seeking approval in the U.S. for…
Reduced thickness of the peripapillary retinal nerve fiber layer (RNFL) in the eye’s retina is associated with worse disease severity…
People who have interruptions in a specific part of their GAA repeats in the FXN gene experience the onset of…
In addition to three recruiting clinical studies that were announced in June, an additional trial is seeking participants with…