About Duchenne - Our Story

Our son, Dezi, received a diagnosis of Duchenne Muscular Dystrophy (DMD) shortly after his 2nd birthday. In short, Duchenne is the most severe and progressive form of Muscular Dystrophy that affects the skeletal muscles, heart & lungs. This genetic disease does not allow for Dezi's body to produce protein to stabilize & rebuild muscle tissue and as it progresses, it will affect his ability to walk, move, and for his heart and lungs to properly function for him to live a healthy and long life. 

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We found Parent Project Muscular Dystrophy shortly after receiving this diagnosis and the resources and advocacy we have received and witnessed from this group have been invaluable to our family as we navigate Dezi's complex health-related concerns. Because of the funding raised for research in treatments for DMD there have been numerous big wins that have directly benefited Dezi.

 

Dezi is currently enrolled in a clinical trial through University of California-San Diego receiving an experimental drug that we hope will result in an improved outcome in exon-skipping therapy treatments for boys with his genetic mutation. As of late 2023, the FDA approved the first gene therapy treatment for people with Dezi's mutation that will deliver a shortened version of a protein into his system to hopefully delay its progression. We just received approval for this treatment as of March 2023 and are expecting to have this procedure done in the summer of 2024. 

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We are so grateful for the advancements in medicine that provide these opportunities to Dezi and our family. This would not be possible without YOUR SUPPORT....AND there is still more work to do.

 

Dezi's treatments are NOT A CURE and there are more people (mostly boys) affected with DMD and other muscular dystrophies that need our CONTINUED efforts to help fund research for treatments.