As many of you know, our son Bradley was diagnosed with Duchenne muscular dystrophy in November of 2000 at the age of 3. We were devastated to hear our son had a life threatening diagnosis that would affect every single muscle in his body including his heart and breathing muscles.We were told he would stop walking by age 12, but he is still walking today at the age of 20. What a blessing this is!
Even though he is still walking, it is not easy. His gait is slow and he tires easily. Steps and inclines are difficult. He needs a wheelchair for long distances. He deals with weakness, fatigue, and pain everyday. In addition, Duchenne has caused him to have decreased heart function and sleep apnea. .
Bradley has always been hopeful for a cure. We have been praying for a cure since his diagnosis. We are very hopeful and excited about all the strides in research and believe the answer to our prayers could be right around the corner.
Dr. Kevin Flanigan and his team at Nationwide Children's Hospital have been working diligently on research that will help Bradley and others suffering from Duchenne muscular dystrophy. Dr. Flanigan has been interested in the ability to skip exon duplications. Bradley lives with a duplication of exon 2. Dr. Flanigan’s lab created a new Duchenne mouse model that contains a duplication of exon 2. Exons are pieces that are linked together to form the gene. Exon skipping alters the pattern in a gene containing a Duchenne mutation in order to restore the reading frame. This gives a patient with Duchenne a slightly different, but still functional dystrophin protein. Pre-clinical data with the mouse has shown that they can restore the skeletal muscle function.
In order to get Dr. Flanigan's research into clinical trials, we need to raise $800,000 to fund a toxicology study and biodistribution study. These funds will allow completion of all pre-clinical data. These final pre-clinical studies need to be completed before moving into the human clinical trial. All of the money donated here will go to Dr. Flanigan's research for this study.
More information about Dr. Flanigan’s research can be found at www.CureDuchenne.org/Flanigan