The recent approval of SKYCLARYS (omaveloxolone) to treat FA in individuals aged 16 and older is a huge step forward for the FA community. Omaveloxolone is a drug that activates a protein that helps the body fight off harmful substances. It shows promise in reducing neurological symptoms and slowing down the progression of the disease, but we still don’t know much about how it affects heart disease in FA. This project aims to fill in this important knowledge gap. Associate Professor Lim and his team want to find out exactly how omaveloxolone affects the cells in the hearts of people with FA. To do this, they will be studying heart cells grown from stem cells of FA patients. They will also use a 3D model of a FA heart, which includes different types of cells found in the heart, to see how omaveloxolone might indirectly affect heart cells through nearby non-heart cells. Additionally, they will also test if omaveloxolone works well with other experimental drugs currently being tested for FA. Their goal is to gather important information on the effect of omaveloxolone on the heart disease in people with FA.
Keith Michael Andrus Cardiac Award | Cardiac Research
Evaluating the cardioprotective effect of omaveloxolone on Friedreich ataxia heart disease
Grant Awarded | Sep 2024
Shiang Lim, MPharm, PhD
St Vincent's Institute of Medical Research
Active
The FARA Grant Program is proud to award the Keith Michael Andrus Cardiac Award to Shiang Lim, MPharm, PhD, of St Vincent’s Institute of Medical Research to study the effect of omaveloxolone on a heart-in-a-dish model of the disease.
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