Opinion|Videos|March 26, 2025

Emerging Treatment Landscape and Future Outlook for Friedreich Ataxia

Author(s)David R. Lynch, MD, PhD

An expert discusses how vatiquinone targets Friedreich ataxia in patients with balance issues by inhibiting 15-lipoxygenase to reduce oxidative stress. Novel dual-route gene therapy combines systemic IV and targeted dentate nuclei delivery, addressing both central and peripheral manifestations while potentially offering disease-modifying benefits.

Video content above is prompted by the following:

A new drug application has been submitted for vatiquinone, an emerging 15-lipoxygenase inhibitor drug, for its treatment effects on upright stability in patients with Friedreich ataxia.
Please discuss the targeted patient population, quality-of-life data, and safety profile for vatiquinone.
The FDA also recently provided clearance for a first-in-industry dual route of administration of gene therapy via systemic IV infusion and direct intradentate nucleiinfusion.
Please briefly explain the rationale of this dual-route administration.
What are the potential implications of gene therapy, both as monotherapy and in combination with other therapies, in the treatment of Friedreich ataxia in the future?
Please share your final thoughts on the treatment of Friedreich ataxia and the future outlook for its treatment.

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