What is Duchenne Muscular Dystrophy?
Duchenne is the most common fatal, genetic childhood disorder, which affects approximately 1 out of every 3,500 boys each year worldwide.
Our Story


After a period of grief and mourning, we managed to gain the strength and courage to learn about Duchenne and meet other Duchenne families.

In our research, we learned two things:

  1. There has never been a survivor of Duchenne
  2. For the first time, there have been some scientific breakthroughs which gave us some hope. We decided to turn adversity into strength.

Currently, Alexander is a typical boy who loves playing basketball with friends and loves school and wants to grow up to be a basketball player.  He is a sweet-natured, fun-loving boy who loves Star Wars and Super Hero movies. Alexander has a special gift that makes people smile and he just radiates light.

Alexander wears boots on his legs at night and knows that they help his muscles. He calls the boots his “Warrior Boots” because they are what Alexander The Great wore. We pray they will make him strong and victorious just like his namesake. Watching him play and run is not something we take for granted. We know what could lie ahead for him, and that is what fuels us to take action.

We founded Walking Strong to solidify our determination and commitment in making our son and other Duchenne boys – the first ever survivors. Duchenne parents are living on a time clock. As most parents make plans and look forward to their children’s future, Duchenne parents, fear the future. We fear what lies ahead for our boys. The time is now – for breakthrough treatments for these boys.

It is our hope and determination that Alexander and other Duchenne boys continue Walking Strong.

Our Mission

Our mission is to build a supportive community to help us end Duchenne Muscular Dystrophy by raising money for ground-breaking research, spreading awareness, and providing aid to children and families with Duchenne

We are hopeful and determined that our boys continue Walking Strong.

What is Duchenne?
(Pronounced “Du shen”)

Duchenne is the most common fatal, genetic childhood disorder, which affects approximately 1 out of every 3,500 boys each year worldwide. It currently has no cure. Diagnosis occurs around 3 – 5 years of age, usually the first sign is muscle weakness and large calf muscles.

The Duchenne gene is found on the X- chromosome and occurs mostly in boys which progressively degenerates all their muscles over time including the heart and the lungs. It is carried through the mother and often times is a spontaneous mutation and does not run in the family. Duchenne does not discriminate and occurs within any racial, ethnic or economic background.

 

The Hard Facts:

  • Duchenne Boys make little or no Dystrophin protein which is responsible for muscle strength and function.
  • Most Duchenne boys are in a wheelchair between 10-12 years old.
  • Duchenne is associated with respiratory failure, heart failure, and debilitating orthopedic complications.
  • Up until just few years ago – upon a diagnosis of Duchenne, Neuorologists offered no hope for the families and told them just go home and love their boys as long as possible.
  • Most Duchenne boys don’t live past their late twenties.
  • There has never been a survivor

 

Current Treatments:

  • In September of 2016, the first ever drug for Duchenne was approved in the U.S.  by Sarepta Therapeutics.  This exon-skipping drug will help 13% of the DMD Population.  The circumstances of the approval at the FDA were highly unusual, and included sharp internal protests that were ultimately overturned.
  • Steroids is the only therapeutic drug prescribed for 100% of Duchenne genetic mutations in order to provide strength and help to slow the progression of the disease.  It is no way a cure, and has many long-term side effects.
  • In February 2017, the steroid drug called Emflaza was approved in the U.S., so parents no longer have to order this superior steroid to prednisone, from the U.K.  
  • Daily stretching by the caregivers and the use of braces at night on their legs, is a necessity for boys with Duchenne.
  • Some boys need surgery to release the tight muscle in their leg as well as spinal surgery in their early teens.
  • Many of the vital needs of Duchenne boys and their families are not covered by health insurance such as:  Power wheelchairs, specialty scooters, night splints for their legs, specialized physical therapist, in-home needs for adaptability such as ramps and stair lift, as well as a handicap accessible van.
  • Many Duchenne boys have characteristics of Autism, ADHD, OCD and anxiety. 
  • Duchenne takes a toll on the entire family.

 

A Ray of Hope:

For the first time in history, there are treatment drugs in the pipeline and clinical trials are happening. Duchenne will not be cured with one therapeutic approach. It will be a cocktail of treatments to dramatically slow the progression of the disease in hopes of making it a chronic illness. Boys with Duchenne have different genetic mutations which makes it complex and some therapies are mutation specific.

WALKING STRONG is supporting Dr. Kevin Flanigan at Nationwide Children’s Hospital in Columbus, OH who is working on a breakthrough exon-skipping drug for Alexander’s very rare mutation of Duplication of Exon 2. Dr. Flanigan’s ground-breaking work could be available in patients in a clinical trial the beginning of 2018.

      1. CRISPR/Cas9 is a naturally occurring reaction that bacteria use to fight viruses. In 2012, scientists discovered they could adapt the process to make cuts in specific human DNA sequences. One part of the CRISPR/Cas9 system acts like a navigation system and can be programmed to seek out a specific part of the genetic code — a mutation, for example. The second part of the system can cut mutations out of the genetic code, and in some cases can replace the mutation with a normal genetic sequence. This technology could eventually be effective for all genetic diseases.
      2. Exon-Skipping: Exon skipping is a way to help make the genetic code readable again, allowing the body to make a shorter form of dystrophin that still works but is mutation specific. Clinical trials are on-going for two different exon-skipping drugs for the most common mutations which is skipping Exon 51 which is around 13% of the DMD population.
      3. Gene Therapy: Gene therapy approaches offer the possibility of delivering a functional copy of the dystrophin gene to muscle cells where it could restore production of the dystrophin protein.
      4. Dystrophin replacement – Duchenne muscular dystrophy is caused by mutations in the dystrophin gene which contains the instructions for making the dystrophin protein- an important component of the structure of muscle. It is thought that another protein called utrophin may be able to compensate for the lack of dystrophin in boys with Duchenne muscular dystrophy since both proteins are structurally similar and appear to have very similar functions.

For more information on Duchenne Muscular Dystrophy please go to:

Website Banner Family Picture

Contact Us

Please fill out the form below with any questions or concerns and we will get back to you shortly.

Not readable? Change text. captcha txt

Start typing and press Enter to search