Theresa Zesiewicz, MD, discusses how growing clinical trial activity, including gene and protein replacement approaches, may help move Friedreich’s ataxia research forward.
Transcript
I think that Friedreich’s ataxia is leading the way in terms of clinical trials in ataxia. We have a tremendous number of Friedreich’s ataxia clinical trials starting.
For example, we have the new Lexeo Friedreich’s ataxia clinical trial for gene therapy, the first of its kind Phase 1, that ended last year.
And preliminarily, it looks like it has very good results, as well as several new Friedreich’s ataxia clinical trials starting. Solid Biosciences, for example, is gene therapy as well, and there will be others starting.
We also have the Larimar study on nomlabofusp that shows protein replacement that had favorable Phase 2 results as well.
So there’s good detection now in ataxia. We’re set up to do clinical trials and to succeed in clinical trials, as well. So I think early detection, clinical trials, great biomarkers are going to help us get to the finish line.