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Givinostat: Hopeful Results in the Fight Against Duchenne

27 Feb

2025

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Duchenne muscular dystrophy is a devastating muscle disease for which there is currently no cure. Fortunately, there is hope thanks to new developments in drug research. One of the promising treatments is Givinostat, a drug that reduces inflammation and promotes muscle recovery. Recently, Givinostat was approved in the United States, and promising studies are also underway in Belgium. Our son, Lander, has been participating in this research for some time, and we would like to share our experiences.

What is Givinostat?

Givinostat is a histone deacetylase (HDAC) inhibitor developed to slow the progression of Duchenne muscular dystrophy. The drug reduces muscle inflammation and helps preserve muscle mass. This can help children with Duchenne remain mobile for longer and slow down the loss of muscle strength.

Promising research results

The effectiveness of Givinostat has been demonstrated in multiple clinical studies. In a large international study involving ambulant boys with Duchenne, participants who received Givinostat showed less deterioration compared to the placebo group. This was evident in tests such as the 6-minute walk test and the North Star Ambulatory Assessment (NSAA). The U.S. Food and Drug Administration (FDA) has approved Givinostat under the brand name Duvyzat® for use in boys aged six and older. The approval process in Europe is still ongoing.

Lander’s participation in the study

Our son, Lander, has been part of a Givinostat study for quite some time. This gives us hope, as research shows that the drug helps slow muscle loss. While we do not expect miracles, we see how important it is to support and participate in these studies. It offers perspective for Lander and many other boys living with Duchenne.

What does this mean for the future?

Further research is still being conducted in Belgium, including at the University Hospital Leuven (UZ Leuven). Here, Givinostat is being tested on both ambulant and non-ambulant boys with Duchenne. The approval in the U.S. is a major step forward, and we hope that Europe will soon follow.

Through these developments, we continue to fight for a better future for Lander and all other boys with Duchenne. We closely follow the studies and hope for further breakthroughs.

Together, we keep fighting for a world with better treatments and, hopefully, one day, a cure for Duchenne!