Voyager Therapeutics has selected a lead gene therapy candidate to advance into clinical trials with the aim of correcting…
Articles by Steve Bryson, PhD
Gene therapy safely reversed heart problems, lowered blood markers for heart muscle damage, and extended survival in a…
A deficiency in the frataxin protein, which is the underlying cause of Friedreich’s ataxia (FA), alters a process in…
Noninvasive diffusion MRI (dMRI) captured changes in the structure of the brain and brainstem of people with Friedreich’s ataxia…
Supplementation with butyrate, a short-chain fatty acid produced in the gut by good bacteria, reversed many diabetes-like metabolic defects found…
Worse ataxia at an initial assessment predicted poor outcomes after four years in people with Friedreich’s ataxia (FA), according…
Researchers have developed a new mouse model of Friedreich’s ataxia (FA) that captures coordination and muscle strength impairment seen…
MRI scans detect large- and small-scale changes in various areas of the brain and brainstem in the early stages…
Continuous exposure to low levels of oxygen, or hypoxia, prevented or rapidly reversed motor symptoms in a mouse model of…
After nearly 1.5 years of treatment, vatiquinone (PTC-743) was found to slow disease progression by 75% in children and…