Researchers have identified a more high-throughput genetic sequencing method to quantify the GAA repeats in the FXN gene that cause…
Articles by Lindsey Shapiro, PhD
A new mouse model of Friedreich’s ataxia (FA), designed to better reflect a more severe disease course, could help…
Skyclarys (omaveloxolone), the first approved therapy for Friedreich’s ataxia (FA), demonstrate a favorable heart and liver safety profile…
Reata Pharmaceuticals’ omaveloxolone has won approval from the U.S. Food and Drug Administration (FDA) for treating Friedreich’s ataxia…
Prime Medicine’s gene-editing technology — called Prime Editing — successfully corrected the genetic deficits associated with Friedreich’s ataxia (FA)…
Low levels of the frataxin protein — the underlying cause of Friedreich’s ataxia (FA) — and the ensuing problems…
Higher than normal activation of the mTOR and AKT signaling pathways — both key to proper cardiovascular function — may…
A combination of the antioxidant resveratrol and the multiple sclerosis treatment dimethyl fumarate (DMF) increased the activity of the FXN gene…
Both clinical severity and frataxin protein levels correlate with the number of disease-causing trinucleotide repeats in the FXN gene of people…
The annual Friedreich’s Ataxia Awareness Month has commenced to raise disease awareness and honor the 15,000 people living with…