Friedreich ataxia (FA) is a rare, hereditary neurodegenerative disease that causes difficulties with coordination and balance and reduces lifespan. The disease affects approximately 1 in 50,000 people ...

A health care provider outlines treatment objectives and supportive interventions for managing Friedreich ataxia, addressing associated comorbidities and symptoms in clinical settings. A key opinion ...

A Prescription Drug User Fee Act target date of August 19, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) ...

Please provide your email address to receive an email when new articles are posted on . Skyclarys was approved in the U.S. for Friedreich’s ataxia in 2023. Approval was based on positive results from ...

Please provide your email address to receive an email when new articles are posted on . Vatiquinone inhibits 15-Lipoxygenase, which regulates the pathways that Friedreich’s ataxia disrupts. PTC ...

Skyclarys activates the Nrf2 pathway which is involved in the cellular response to oxidative stress. The Food and Drug Administration (FDA) has approved Skyclarys ™ (omaveloxolone) for the treatment ...

The FDA approved omaveloxolone (Skyclarys) for the treatment of Friedreich's ataxia in patients ages 16 and older, making it the first approved treatment for this rare progressive neurodegenerative ...

Solid Biosciences announces FDA clearance for SGT-212, targeting Friedreich’s ataxia with dual gene therapy administration routes, initiating Phase 1b trial in 2025. Solid Biosciences Inc. announced ...

The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for ...

Friedreich’s ataxia is an ultra-rare, inherited neurodegenerative disorder that is typically diagnosed during adolescence. Patients with Friedreich’s ataxia experience progressive loss of coordination ...

The US Food and Drug Administration (FDA) has approved the first treatment for the neurodegenerative disorder Friedreich's ataxia for use in adults and adolescents aged 16 and older. The recommended ...