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DUCHENNE Muscular Dystrophy is one of the most common fatal genetic disorders to affect children around the world. Approximately one in every 3,500 boys worldwide is afflicted with Duchenne Muscular Dystrophy with 20,000 new cases reported each year in the developed world.
It is a devastating and currently incurable muscle-wasting disease, associated with specific inborn errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fibre function. Symptoms usually appear in male children before the age of five. Progressive muscle weakness of the legs and pelvis eventually spreads to the arms, neck, and other areas. By age 10, braces may be required for walking, and most patients are confined to a wheelchair by age 12.
Eventually, this progresses to complete paralysis and increasing difficulty in breathing, requiring ventilation. The condition is terminal, and death usually occurs before the age of 30.
The outpatient cost of care for a non-ambulatory DMD boy is among the highest of any disease. There is currently no cure for DMD, but for the first time ever, there are promising therapies in or moving into clinical development.
WE need as many people to join Jack in his fight as possible - what can you do to help?
Here at Joining Jack, we are grateful for every little thing that you can do to help support our cause. Its people who participate in events to help raise money and awareness for the charity who are helping us make a massive difference in our fight to find a treatment for Duchenne. Joining Jack are making huge advances in research to eventually find a cure for this horrible disease, your donations go straight into the hands of carefully selected doctors and researchers who are very close to finding a cure for so many sufferers.
For more information about DMD visit www.treat-nmd.eu. If you or a family member suffers from DMD, please consider registering your details at The DMD Registry for further information on and access to current clinical trials.