An international study to learn about your preferences for DMD Treatments

La Force DMD is seeking your participation in a research survey for parents or guardians of sons with Duchenne muscular dystrophy and adult men (18+ years) with Duchenne. This is an international survey about your preferences and priorities for Duchenne treatments.

Regulatory agencies such as the EMA and FDA rely on the input of patients, families, and drug approval examples from other countries to inform treatment approval decisions. This preference survey will be the first study to measure preferences for Duchenne treatments internationally. The findings from this survey will be returned to participating patient advocacy organizations and may be used to inform policymakers about what you want out of treatments, and what risks you are willing to take.

The goal of the survey is to learn about your preferences and priorities for Duchenne treatments and to see if preferences vary internationally. Duchenne patient and advocacy organizations from Australia, Belgium, Canada, the Netherlands, the United Kingdom, and the United States have come together along with researchers at Johns Hopkins University to conduct this survey.

This survey is about your preferences and priorities for Duchenne muscular dystrophy treatments. This is the first study to measure Duchenne preferences internationally. DMD advocacy organizations globally have come together to launch this survey, and results will be returned to patient advocacy organizations and used to inform policymakers about what you want out of treatments, and what risks you are willing to take.

Survey info

  • Online
  • 45-60 minutes
  • No payment
  • Some questions might make you feel upset.
  • Treatments described include risks of severe side effects.
  • It is your choice to participate
  • You can stop the survey at any time
  • Results will be anonymous

Who can participate

  • Male adults (18 years+) with DMD
  • Parents of living sons with DMD
  • People currently living in Australia, Belgium, Canada, Netherlands, UK, or the US
  • Don’t match this description? Reach out, and we will accommodate

This research survey is online and will take 45-60 minutes. You will not be paid for completing the survey. It is possible that some of the questions in this survey might make you feel upset. The treatments that we describe include risks of serious side effects. It is your choice to complete the survey. You can stop the survey at any time. The results of the survey will be anonymous.

To participate, please email the survey@jessesjourney.com

If you have questions or concerns, please contact Norah Crossnohere at ncrossn1@jhu.edu

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Duchenne Awareness Day

Today, September 7th is International “Duchenne Awareness Day.” We took this opportunity to launch our video. Its aim is simply to raise awareness about Duchenne muscular dystrophy.

The seventh day of the ninth month represent the 79 exons in the dystrophin gene.

Telling as many people as possible about Duchenne muscular dystrophy (DMD) is the first step towards a cure. 

Duchenne muscular dystrophy is a degenerative disease of the muscles. It affects children at a very young age (mostly boys). Causing a weakening of every muscle in the body, it leads to loss of mobility, heart problems, respiratory failure and premature death of those afflicted in their early twenties. There is currently no cure.

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 Foundation, 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 participating 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.

 

Share this video and Donate here.

More about Duchenne Muscular Dystrophy (DMD)

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 of him, and he’ll use 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…

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