Our vision is our name:
CureDuchenne

CureDuchenne was founded by Paul and Debra Miller in 2003 after their son was diagnosed with Duchenne muscular dystrophy. The Millers applied their professional backgrounds to create a fiscally responsible and strategically focused business model, and assembled a seasoned staff and an expert Scientific Advisory Board, to lead the charge for a cure. Today, our nonprofit is a national leader in affecting change worldwide.

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What is Duchenne?  

Duchenne is a devastating muscle disease in children. Historically, most boys who have it do not survive beyond their mid-twenties, and those that do will be wheelchair bound by age 12 and experience social isolation. The simplest of tasks become difficult, and in the later stages, heart and breathing muscles begin to fail. Nearly 20,000 boys are living with the disease in the United States alone and over 300,000 worldwide. The disorder knows no cultural, economic, or social boundaries.

Muscular dystrophies are a group of genetic disorders characterized by muscle weakness. Duchenne is the most common and severe form, caused by loss of dystrophin, beneath the sarcolemna. The molecular mechanisms of the disease have been extensively investigated since the discovery of the gene in 1986.

The good news is that new gene-based therapies have recently emerged with noted advances in using conventional gene replacement strategies, RNA-based technology, and pharmacological approaches. In particular, antisense-mediated exon skipping has shown encouraging results and holds promise for the treatment of dystrophic muscle.

Cletki
Initial signs of cardiac dysfunction caused by Duchenne muscular dystrophy are usually detected during adolescence. However, decreased physical activity, even if wheelchair-bound, can allow better tolerance of decreased cardiac function.
 

Mission and Vision  

Our efforts are targeted. We have the leading scientists in the world helping us determine the most viable research projects, accelerate the clinical trial process and bring potential life-saving drugs to help this generation of Duchenne boys.

Our vision is our name…to cure Duchenne muscular dystrophy.

Our mission is to save this generation of Duchenne boys.

With increased awareness and increased funding we’ll extend and improve the lives of those afflicted with Duchenne.

 
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The Words of a Duchenne Parent  

I was so excited when I found out I was expecting a baby and going to be a mother for the first time. When I saw him for the first time, I just fell in love with my perfect little baby boy, Ian Michael Schneider. He was such a great baby – he slept, ate, laughed and had the chubbiest cheeks and the brightest eyes.

Three years later, our lives changed forever in a matter of an hour when my husband, Michael, and I were thrown into the world of Duchenne Muscular Dystrophy. We learned Ian will not live a full life and our family will have to watch him suffer as the disease progresses.

I often think, “I did this to Ian.” Not intentionally, I know, but I still passed it to him.

Ian just turned 9 and things are very different for him and the rest of our family. Instead of being fitted for a football uniform, he is being fitted for a motorized scooter. I hate to watch Ian frustrated when he falls down, when he struggles in school with physical and academic issues, when he can’t do anything but be carried on daddy’s shoulders at his cub scout camping weekend, when he has no one but us to play with, when he has to sit in a wheelchair on his class trip because there is walking involved.

My heart breaks when I have to give him a growth stunting steroid every day, when we feel out of place among healthy families or shut out completely because they can’t relate, when we dread the day the words “I can’t walk anymore” will come out of Ian’s lips, when the only thing on my mind is how long will we have our son and when will Duchenne take everything from him and our family?

By Donna Schneider, Phillipsburg, New Jersey

 
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Message from the Founders  

Imagine having a beautiful baby boy. For four years, your child thrives and is the light of your life. One day, you notice he has difficulty standing up. His calves are swollen, and he can’t keep up with other children. You take him to his pediatrician, who says he’s probably just a slow starter and not to worry.

At five, you finally find a specialist that gives a proper diagnosis: Duchenne muscular dystrophy. You’re told his muscles will die and not regenerate. You scream frustration as to why you haven’t heard about Duchenne before. Then, you find out pharmaceutical companies do not invest in it because the number of potential users does not make it profitable enough.

That’s our experience, and for the love of our child, we’ve taken matters into our own hands and embarked on a path to affect change: to raise the funds needed to find a cure within our son’s lifetime. We’ve always had faith, but we never knew how much we would come to rely on it. We hope all Duchenne families are provided the comfort needed to get through these difficult times.

We are thankful to our partners and sponsors for sharing our vision and collaborating with us to find a cure. Saving the children affected by Duchenne has become our life’s work, and we hope you’ll join with us.

Paul and Debra Miller
Founders, CureDuchenne

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