Opinion
Video
Emerging Treatment Landscape and Future Outlook for Friedreich Ataxia
Author(s):
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.
Related Videos
Related Content
Published: March 27th 2025 | Updated: March 28th 2025
FDA Approves Diazoxide Choline Extended-Release Tablets for Hyperphagia in Prader-Willi Syndrome
