Cure for Cole
Donation protected
Hi! I’m Cole. I’m 7 years old and I have a form of Muscular Dystrophy called Duchenne. Duchenne Muscular Dystrophy is the #1 genetic killer of young children and affects one in every 3,500 boys born worldwide. Currently there is NO cure and the disease is 100% fatal. There are no survivors. My muscles are missing a protein called dystrophin, which supports the muscle fibers, keeping them strong. When a normal muscle that is being exercised would get stronger, mine gets weaker. The natural course of the progression of the disease will have me wheelchair bound by 9 – 12 years of age, experiencing heart and lung problems next by age 15 or so and generally death will follow by late teens to early 20’s. It is an especially brutal and cruel disease. I have to take nasty tasting steroids for life to help slow the loss of muscle strength and I also go to Physical and Occupational Therapy at CHKD each week. I just finished my 2nd year of Preschool where I received Special Education and additional Physical, Occupational, and Speech Therapy services. I wear braces to bed at night and I am slowly adjusting to the idea of having to wear braces during the day too. I get tired easily and it’s hard for me to keep up with the other kids on the playground. I face many challenges daily like needing help to get up and down steps, and running and jumping are difficult or nearly impossible in some situations. I have just started to be able to ride a tricycle although I do need an extra push here and there to help me get going. I especially enjoy riding my motorized four-wheeler. I love feeling that wind to my face!
Research has made huge progress over the last 10 years and there are now 3 drugs that are in clinical trials that are looking very promising for the Duchenne community. With the FDA considering these New Drug Applications, my family and I are hopeful that a cure may be found in time to save me. The doctors say that my generation of boys with Duchenne may be the last to die or the first to survive….. and I want to LIVE!!!
Cincinnati Children’s Hospital Medical Center has a Neuromuscular center run by Dr. Brenda Wong, who is known throughout the Duchenne community as one of the top specialists of DMD in the country. He will be seen by Neurology, Cardiology, Pulmonology, Endocrinology, Neuropsych, have Physical, Occupational, and Speech Therapy evals, Social Work, Care Management, and a Nutrition consult. He will also have multiple lab draws and radiology studies such as an EKG, Echo, DEXA Scan, and a Holter monitor for 24 hours. They will then gather all the information together and develop a treatment and care plan and adjust his medications for him, hopefully with the goal to get him into a clinical trial. As his mother, I want him to get top notch care and treatment, giving him the best chance to live. I believe Cincinnati will give him that chance. This trip is costly and we are anticipating follow up appointments every 6 months as well as his regular appointments at home in Virginia, along with the mounting medical bills, co-pays, therapy, and medical equipment costs- and that is why I am asking for help. Cole’s father passed away from suicide when he was only 8 months old. As if that wasn’t devastating enough, Cole was diagnosed with Duchenne almost exactly 2 years later. It has been a heartbreaking last few years along with financially burdensome. Any help is appreciated. I just want to give my child a full, happy life, for as long as God will let me have him!
It only takes one muscle to make a difference- your heart!
Research has made huge progress over the last 10 years and there are now 3 drugs that are in clinical trials that are looking very promising for the Duchenne community. With the FDA considering these New Drug Applications, my family and I are hopeful that a cure may be found in time to save me. The doctors say that my generation of boys with Duchenne may be the last to die or the first to survive….. and I want to LIVE!!!
Cincinnati Children’s Hospital Medical Center has a Neuromuscular center run by Dr. Brenda Wong, who is known throughout the Duchenne community as one of the top specialists of DMD in the country. He will be seen by Neurology, Cardiology, Pulmonology, Endocrinology, Neuropsych, have Physical, Occupational, and Speech Therapy evals, Social Work, Care Management, and a Nutrition consult. He will also have multiple lab draws and radiology studies such as an EKG, Echo, DEXA Scan, and a Holter monitor for 24 hours. They will then gather all the information together and develop a treatment and care plan and adjust his medications for him, hopefully with the goal to get him into a clinical trial. As his mother, I want him to get top notch care and treatment, giving him the best chance to live. I believe Cincinnati will give him that chance. This trip is costly and we are anticipating follow up appointments every 6 months as well as his regular appointments at home in Virginia, along with the mounting medical bills, co-pays, therapy, and medical equipment costs- and that is why I am asking for help. Cole’s father passed away from suicide when he was only 8 months old. As if that wasn’t devastating enough, Cole was diagnosed with Duchenne almost exactly 2 years later. It has been a heartbreaking last few years along with financially burdensome. Any help is appreciated. I just want to give my child a full, happy life, for as long as God will let me have him!
It only takes one muscle to make a difference- your heart!
Organizer
Mandy Williams
Organizer
Suffolk, VA
