112: Exploring Dimethyl Fumarate to Treat Friedreich Ataxia

NeurologyLive® Mind Moments®

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In this episode, Francesco Saccà, MD, PhD, an associate professor of neurology at the University of Naples, discussed an ongoing study assessing the use of dimethyl fumarate, an approved therapy for relapsing multiple sclerosis, in patients with Friedreich ataxia (FA). He spoke on the mechanism of action of the agent and why it can be beneficial in this patient population, as well as how it differs from omaveloxolone (Skyclarys; Biogen), the first approved treatment for FA. In addition, he outlined the study design, the primary end point of frataxin decrease, and what would be considered a "successful" study. Furthermore, he spoke on the reasons behind the trial and what led investigators to this point.
Looking for more ataxia discussion? Check out the NeurologyLive® ataxia clinical focus page.
Episode Breakdown:

• 1:05 – Origin of the study
• 3:00 – Conduct of the study, end points, goals
• 5:10 – Promising mechanism of action of dimethyl fumarate to treat Friedreich ataxia
• 7:30 – Questions in clinical impact of changes in frataxin
• 10:00 – Neurology News Minute
• 12:40 – What is considered a successful study?
• 14:55 – Patient inclusion, demographic makeup of the study
• 16:35 – Timeline of the trial and data readout

The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here:

• FDA Approves Alexion's Ravulizumab-cwvz for Neuromyelitis Optica Spectrum Disorder
• Epilepsy Agent STK-001 Demonstrates Disease-Modifying Effects in Early Phase Studies of Dravet Syndrome
• Eisai Submits sBLA for Monthly Intravenous Maintenance Dosing of Alzheimer Therapy Lecanemab

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