After following a group of patients with a specific gene mutation for many years, a team of UC Davis MIND Institute scientists has provided important insights into how fragile X-associated tremor/ataxia syndrome (FXTAS) first develops. The work, led by researchers David Hessl and Susan Rivera, identifies new ways to study the disease and possibly test potential therapies in the future. FXTAS, caused by «premutation» expansions of the FMR1 gene, has no approved treatments, only symptomatic management. The study was published in the Journal of Neurodevelopmental Disorders.
New insights into FXTAS could inform future research and clinical trials
After following a group of patients with a specific gene mutation for many years, a team of UC Davis MIND Institute scientists has provided important insights into how fragile X-associated tremor/ataxia syndrome (FXTAS) first develops. The work, led by researchers David Hessl and Susan Rivera, identifies new ways to study the disease and possibly test potential therapies in the future. FXTAS, caused by "premutation" expansions of the FMR1 gene, has no approved treatments, only symptomatic management. The study was published in the Journal of Neurodevelopmental Disorders.