Meet Tyler James Ash, our rambunctious, fun-loving 5 year old. He has an infectious smile and personality that lights up the room. Although Tyler looks like your regular preschooler on the outside, he's fighting a major battle on the inside. Our sweet perfect boy was diagnosed with Duchenne Muscular Dystrophy at the very tender age of 3. This progressive disease will begin to strip away his very ability to walk, run, jump, breathe, eat and even give his mom a hug. This disease knows no boundaries and will continue to destroy other muscle groups including his heart and lungs. The onset of these symptoms will begin in just 5 short years. Most boys will begin to lose their ability to walk around the age of 10 followed by full-time wheelchair use in the mid-teens. It's 100% terminal with a life-expectancy around the age of 25. These are not easy statistics to process as a parent. Looking into his future is just a grim reminder that our time together is limited. Knowing your child is weakening every year makes each birthday painfully bittersweet. This is the path that he would inevitably be faced with until now.
Tyler has an opportunity to be spared from the devastation of DMD. He has a duplication of exon 2 on the dystrophin gene. Currently, Dr Kevin Flanigan's team at Nationwide Children's Hospital is doing amazing work with exon 2 duplications and have developed a treatment specific for this mutation. This is the answer we've been praying for! This treatment would not only help Tyler but other boys with mutations within the first few exons. This could change the course of this rare disease for a community of children. An opportunity that I never thought existed. Tyler's uncle passed away from the very same disease in 2005. It was a heartbreak that I thought I would never have to endure again, but I was so very wrong. Here we are now in the greatest fight of our lives with our own son.
The drug we need that could save his life is currently in a lab waiting for funding. The current need is to raise $800,000 to fund a toxicology study and biodistribution study. The funds will allow completion of all pre-clinical data. These final pre-clinical studies need to be complete before moving into human clinical trial. All of the money donated here will go to Dr. Flanigan's research for this study. The treatment is there waiting. We just need your help to get to the finish line.
For additional information on Dr. Flanigan's research, please visit the link below.
Thank you so much for your support!
Chris and Marilyn Ash