BRUCE’S UNBELIEVABLE GIFT TO HELP HIS PATIENT

Bruce Babington, an Osteopath, based in Ste-Agathe-des-Monts, Québec, is taking on the challenge of cycling across Canada in July 2018. He was inspired by his patient, Anakin Lacasse, who is afflicted with Duchenne muscular dystrophy. Why riding across Canada? To spread knowledge about DMD and raise money for research, the only hope for children like Anakin…

Riding across Canada for hope!

Bruce plans to cycle an average of 200 KM from Mont-Tremblant to Vancouver and complete the journey in 24 days. When he first started to treat Anakin, he didn’t know much about DMD, but it became clear to him very fast that there was no quick fix! He promptly decided to offer him free services for life. He also had the idea in the back of his mind that someday he would like to go across Canada by bike. He also learned about Fondation La Force, the organization inspired by Anakin, which helps him and other people afflicted with Duchenne to get closer to a cure. At one point, the idea of cycling across Canada for La Force crossed his mind, and he made the decision. He will be taking one month off in July 2018, with the goal of cycling 200 KM a day and complete the distance between Mt-Tremblant and Vancouver within 24 days.

 

Go big or go home!

“You know, the challenge I’ll face in that 24-day period is nothing like the challenge Anakin is going to face every single day of his life. What I can really do for him is try as much as I can to help promote La Fondation La Force and really spread the knowledge of what Duchenne really is” said Bruce Babington to our team.

 

About 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. It is extremely rare that Duchenne muscular dystrophy (DMD) will 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 typical evolution of the disease inevitably brings about premature death between the ages of 18 and 25.

 

We need your donation:

Our team will work hard to give the visibility Bruce needs to unite people around DMD and raise awareness in the general public about the disease.

Thank you in advance to all of our friends in the DMD community, for encouraging Bruce!

 

Don’t forget, sharing our stories is also an excellent way to support our cause.

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TAKING ACTION PORTRAIT OF DUCHENNE – WHEN LIFE CHANGES

In this inspiring interview of our series “Portrait of Duchenne,” La Fondation La Force talks with Émilie Meeus, Oscar’s mother, raising funds for research into Duchenne muscular dystrophy (DMD). Émilie’s life changed when her three-year-old son Oscar was diagnosed with DMD. She decided to do something to make a difference including by starting an association to help the research.  We interviewed Émilie in 2016 at Action Duchenne conference in London.

 

Before diagnosis

Émilie and her family live in Brussels, Belgium. Before they received the diagnosis, the family had at first to wait for the results, hoping the muscular dystrophy wouldn’t be Duchenne’s. They carried hopes and expectations based on earlier negative tests which were given by doctors.

Émilie’s family was perhaps about to learn a shattering news which would drastically change the direction of their life.

 

After diagnosis

Learning about the diagnosis, the family was immediately broken down knowing about the news: “As we first heart that word, that middle name word (Duchenne), all the world has really fallen apart around us.” Émilie and her family had to go through despair and through all this shocking phase facing this different reality that was involved in living with the disease. After that, things got better, and they were able to move later on: “Fortunately, daily life too over. You begin to understand that life goes on.”

An appointment with another doctor helped and encouraged them to accept the situation, to adapt and to reorganize themselves. This specific doctor simply said: “You’re going to live a different life than others, but you’re going to be happy. And the happier you’re going to be, the happier your son will be because children adapt faster and better than us.”

Once they have accepted the situation and they have been supported by professional doctors, some other things interacted and helped them move on and progress.

 

Watch Oscar’s full story >

To create an association

Émilie Meeus and her family wanted to create an association to raise funds and support Duchenne muscular dystrophy’s research: “We’re going to try to help the research and treat the disease. And, even if we fail it, we’re going to give him the best possible life.” So, besides allowing them to stay well-informed about the disease and its latest developments and so for the progress of the research, they’ve been able to expand their social circles, talk with other people suffering from the same disease and listen to their experiences. Their approach allowed them to stay positive and hold on the hope of living a happy life.

 

Reorganisation and life changes

In the final stages of their adaptation process, Émilie and her family had to accept the imposed limits of the disease to allow them finding satisfaction and joy again despite all the difficulties encountered. For instance, their special condition may include positive aspects such as living way more in the present moment: “Perhaps, we enjoy more daily life than others. Maybe it’s that we project ourselves less into the future. I’ve always tended to try looking far ahead. As for now, I do it less because you can never know… Still, it helps me enjoy whatever day holds, it really does.”

 

To broach the subject with children

Émilie found a simple way to broach the DMD disease with her son Oscar comparing it as a “leg’s boo-boo.” Since children are too young to understand all the details of the disease, at least, it allows them to acknowledge their medical situation and recognize it on other people suffering from the same disease: ” When we met another family, we explained to Oscar that we would see a little boy having the same leg’s boo-boo as him.”

Until Oscar is old enough to understand his medical situation, he’s for now able to figure it out through images and simplification with the help of his family.

 

DMD changes your life in a split second

The progression of DMD is unforgiving: the body’s muscles gradually weaken, leaving children with DMD in a wheelchair by age 12. They have a life expectancy of 20 to 30 years. There is no cure and, in Canada, no treatment for the cause. Two treatments for DMD are approved in the USA and Europe, but they don’t benefit all children with DMD. Promising treatments, in development, are expected within 5 to 10 years.

Emilie’s foundation > Little O against DMD

 

Acknowledgements

We thank Action Duchenne, who received us with open arms to conduct a series of interviews.

  

Action Duchenne

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Solid Biosciences Initiates Clinical Trial for Gene Transfer

Last week we had the privilege to meet in person with the team at their headquarter in Boston. They presented their very robust preclinical trial data suggesting that SGT-001, has the potential to slow or stop the progression of DMD, regardless of genetic mutation or disease stage. Now they are announcing the initiating of SGT-001, their investigational microdystrophin gene transfer for DMD and we are so excited as this might become a life-changing treatment for people with DMD.

 

SGT-001 Clinical trial is an Incredible milestone

This is an incredible milestone for this startup company of four years old. This accomplishment is the reflection of years of ongoing collaboration with the top experts in gene therapy. SGT-001 is based on pioneering research in dystrophin biology by Dr. Dongsheng Duan of the University of Missouri and Dr. Jeffrey Chamberlain of the University of Washington.

 

In case you don’t remember the specifics about the microdystrophin and gene therapy we invite you to watch the interview we conducted last year in London with Dr Jeffrey Chamberlain:

 LINK TO VIDEO

 

About the clinical trial:

The Phase I/II clinical trial, called IGNITE DMD, is a randomized, controlled, open-label, single ascending dose study that will evaluate the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients with DMD.  IGNITE DMD, adaptive in nature, will allow Solid Biosciences to adjust dose and number of patients as the study progresses to efficiently characterize the safety and efficacy of SGT-001. The patient screening will begin at their first participating study in one location in the United States in the coming days. Solid Biosciences is working to bring on additional sites in the United States and abroad.

 

About SGT-001

SGT-001 is a novel adeno-associated virus* (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD. SGT-001 is a systemically administered* candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins.

  • Adeno-associated virus > is a small virus which infects humans and some other primate species. Link

  • Systemically administered > Systemic forms of administration affect the whole body (in general).

 

To know more

Open letter de Solid Biosciences

 If you are interested in learning more about this trial (IGNITE DMD), further information will be available on clinicaltrials.gov shortly.

Gene transfer has the potential to address the genetic cause of Duchenne muscular dystrophy.

Press release