Daily use of the experimental therapy nomlabofusp is leading to long-term clinical improvements and increases in frataxin levels for…
Articles by Lindsey Shapiro, PhD
Using MRI scans, a team of researchers identified several differences in the brain and spinal cord of people with…
The investigational gene therapy LX2006 was well tolerated and led to clinically meaningful improvements in cardiac biomarkers and functional…
CAP-004, Capsida Biotherapeutics’ experimental gene therapy for Friedreich’s ataxia (FA), safely delivered its genetic cargo to key disease-related…
Biogen’s Skyclarys (omaveloxolone) has been approved in Canada to treat people with Friedreich’s ataxia (FA) ages 16 and…
The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to…
Data from wearable sensors that are designed to collect real-world information about a person’s ability to function in daily life…
Inhibiting ferroptosis, a type of iron-dependent cell death, could be a promising therapeutic strategy for heart disease management in people…
Friedreich’s ataxia (FA) patients with less common types of disease-causing mutations in the FXN gene may have a distinct clinical presentation…
A gene-editing therapy designed to correct the FXN gene defect that’s associated with Friedreich’s ataxia (FA) reversed several cellular features…