Today, September 7th is an international “Duchenne Awareness Day.” We took this opportunity to launch our video. It aims to inform the public about Duchenne muscular dystrophy and new experimental treatments.
Telling as many people as possible about DMD is the first step towards a cure.
Hope for new treatments!
For the first time in the history of DMD, treatments that address the causes of the disease are emerging. Some tackle specific genetic mutations. In the case of Ataluren, a treatment presently available in Europe, it targets the so-called “nonsense” mutation. If you have DMD or a loved one is suffering from DMD, it is essential to get a genetic profile.
We need your help to find new treatments for all children afflicted with DMD.
It is through public awareness that, together, we will get closer to a cure. At La Force DMD, we work tirelessly to unite the DMD community to raise awareness around a common objective: providing access to new treatments as fast as possible and participate in the funding of new research projects.
Together, our voices are heard further!
We firmly believe that there is Strength in Unity.
We believe that, together, we can be part of the solution and contribute significantly to finding treatments for all children with DMD.
Help us!
Share this video and Donate here.
DUCHENNE MUSCULAR DYSTROPHY (DMD)
A degenerative disease of the muscles for which there is no treatment
Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. Duchenne muscular dystrophy (DMD) will rarely affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.
The cause of Duchenne Muscular Dystrophy (DMD): A genetic mutation
The simplest way to explain the disorder is that a genetic mutation (i.e., a genetic defect) affects the gene responsible for the production of dystrophin. This genetic defect prevents the gene from synthesizing dystrophin.
The consequence of Duchenne Muscular Dystrophy (DMD): Dystrophin deficiency
Dystrophin plays an essential role in maintaining the integrity of muscle cells. Because of the genetic mutation they harbour, people who have Duchenne muscular dystrophy (DMD) lack dystrophin in their system. In the absence of dystrophin, muscles degenerate and become atrophied.
The effects of Duchenne Muscular Dystrophy (DMD): Gradual loss of the ability to use one’s muscles
The disorder causes muscles to degenerate and atrophy: one by one, all muscles in the body gradually fall prey to the disease. The fate of our child, Anakin, will be a gradual loss of his ability to walk. Around the age of 10, playing baseball, running and walking will be only memories for him, and he’ll be confined to a wheelchair. Throughout this time, he will be aware of this degradation process.
A few years later, during preadolescence, even the ability to take a deep breath may be a thing of the past since the disease ultimately causes respiratory failure. Of course, DMD does not spare the heart (also a muscle) either… Without treatment, the normal evolution of the disease inevitably brings about premature death between the ages of 18 and 25, sometimes earlier. For families, there is no worse sentence than to watch their child suffer helplessly…
