Cycling across Canada to cure DMD

What’s it like to live with DMD?

As a parent, to face the reality that your child has a fatal disease? As a person with DMD, to face an inevitable, premature death in your twenties? As a sibling, relative or friend, to face mourning your loss?

The journey is beyond words.

 

Our Web short documentary series, “Portrait of Duchenne Canada,” will present the experiences of families and children with DMD. You will meet five Canadian families who are taking on the challenges of raising money and leading the way in advocating for access to new treatments. Today, more than ever, it’s time to share their stories. New treatments are on the horizon, and families need to be heard to advocate for ways to access them quickly.

 

This is the story about Bruce Babington, who will cycle across Canada in July 2018 to raise funds and awareness for DMD.

 

A radical decision

“I made rather a foolish decision to ride solo across Canada.” 

-Bruce Babington

 

Bruce has made a radical decision to cycle solo across Canada in July 2018. His goal is to help people afflicted with Duchenne muscular dystrophy (DMD). Bruce is an osteopath. About one year ago, he started to treat Anakin Lacasse, a 9-year-old boy with DMD.  It was the first time that Bruce had encountered a patient with this condition. Anakin was rapidly losing muscle strength, and Bruce quickly became aware of his patient’s reality. Then, he learned about Anakin’s mother’s involvement in La Fondation La Force.

Bruce was so moved that he made up his mind, just like that, to do something to help. So, in July 2018, he’s taking one month off work to cycle an average of 200 km/day between Mont-Tremblant, QC, and Vancouver, BC, in 24 days.

 

First impressions

  • “When I first met Anakin, I was very inspired by him.”
  • “It’s very clear that there’s no quick fix in this instance.”
  • “I wanted to cycle across Canada, so why not do that for La Fondation La Force?”

 

The overriding challenge

Jacques Tremblay, medical researcher (translation): “The main challenge in trying to develop therapies for rare diseases is getting funding to do the research. The direct impact of foundations, such as La Force is that, firstly, they help spread awareness of this disease to the general public. They raise funds that are essential to get preliminary results and then be able to go after larger grants from government-dependent organizations. If we apply for grant applications to those organisations without preliminary results, basically, our chances of having a grant are zero.”

Bruce Babington: “The challenges I have in a 24-day period are nothing like the challenges Anakin is going to have every single day of his life. I try to do as much as I can to help promote La Fondation La Force.”

 

How will cycling across Canada help the DMD community?

  • We are running a fundraising campaign: “Bruce Across Canada.” La Fondation La Force will award all “Bruce across Canada” funds to the most promising Canadian research.
  • We want to give a voice to the Canadian DMD community. Bruce’s endurance challenge is about starting a conversation on access to new treatments in Canada. Two new treatments for DMD are approved elsewhere in the world. Many other treatments are in the final phase of clinical trials. Canada has no framework to review new therapies for rare diseases quickly and efficiently, and the reimbursement process for such treatments is incredibly cumbersome. Add your voice to our conversation. Be a change maker.
  • Help us to ensure that a whole generation of children and young adults with DMD can access new treatments. It’s time to rise to the challenge!

 

Join the fight!

Duchenne muscular dystrophy (DMD) is a rare disease with no cure. Orphan drugs that target its causes are being developed now. But we need your help. Research is the only hope. And research needs funding.

For the past 20 years, Canadian families in the DMD community have been raising funds for and awareness of DMD. Their actions and successes have funded current research. Now that new treatments are emerging, families need to unite and be heard to access new treatments quickly.

 

Our part is to tell their story. Sharing these stories is one step forward towards the cure.

Donate to “Bruce across Canada” Donate here. 

We invite you to share this story with your community.

Donate to our organization, so we can continue to help the DMD community to get closer to treatment. Donate here.

 

For more information:

About DMD: Here

About “Bruce across Canada” Here

About reimbursement of drug costs: Here

Decision from the FDA’s Office for Ataluren

Today, PTC Therapeutics, Inc, announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA’s prior position and denied PTC’s appeal of the Complete Response Letter concerning the New Drug Application (NDA) for ataluren. In its letter, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway.

This would involve a re-submission of an NDA containing the current data on the effectiveness of ataluren with new data to be generated on dystrophin production in nonsense mutation Duchenne muscular dystrophy (nmDMD) patients’ muscles, as quantified by procedures to be agreed upon between PTC and the FDA and using newer technologies. The letter adds that PTC’s Study 041, which is currently enrolling, could serve as the confirmatory post-approval trial required in connection with the accelerated approval framework. In a clarification teleconference with the FDA promptly after receiving the letter, PTC indicated its intent to follow the FDA’s recommendation and preliminarily discussed methods to collect such dystrophin data and expedite this potential path forward.

The patient will still have access to treatment:

Based on these interactions, PTC currently intends to maintain patients in the U.S. currently receiving ataluren for nmDMD through an expanded access clinical program during this process.

About the AdCom watch our video:

 

About Translarna™ (ataluren)

This treatment is the first to target an underlying genetic cause of DMD: a nonsense mutation (nmDMD). It aims to slow the progression of DMD by restoring the function of a specific protein, dystrophin. In DMD, a nonsense mutation corrupts the genetic machinery that builds a working version of this protein, which is essential for proper neuromuscular function.

In clinical trials, ataluren has shown some benefit in slowing the loss of motor skills in some children with nmDMD. Some boys treated with ataluren could more easily undertake a range of physical activities, including walking, climbing and descending stairs and other motor functions.

In 2014, the European Medicines Agency granted Translarna™ (ataluren) conditional marketing authorization to treat nmDMD in children of 5 years of age or more and still able to walk. Translarna™ (ataluren) is also approved for use in South Korea and Israel.

At the end of 2016, Translarna™ (ataluren) received marketing authorization by the European Medicines Agency and is available in Europe and regions that reference that authorization.

On the 25h of October, the Office of Drug Evaluation I of the FDA provided a Complete Response Letter (CRL) for the application to market ataluren in the United States. This means that the FDA believes ataluren cannot be approved at this time. PTC Therapeutics Inc. is planning on filing a formal dispute resolution request next week. The dispute resolution process gives PTC an opportunity to appeal the letter issued by the Neurology division and have the decision on the application reviewed by the Office of New Products within the FDA. PTC Press Release

 

Understanding Ataluren approbation process

For more information

What is the AdCom?
Families at the AdCom September 2017
FDA close the door on Ataluren

Short Docs series: Portrait of Duchenne Canada

Our web short documentary series, “Portrait of Duchenne Canada”, will be coming son! 

We will present the experiences of families and children with DMD across Canada.What’s it like to live with Duchenne muscular dystrophy (DMD)? As a parent, to face the reality that your child has a fatal disease? As a person with DMD, to face an inevitable, premature death in your twenties? As a sibling, relative or friend, to face mourning your loss?

 

The journey is beyond words.

Our web short documentary series, “Portrait of Duchenne Canada”, will present the experiences of families and children with DMD. You will meet five Canadian families who are taking on the challenges of raising money and leading the way in advocating for access to new treatments. New treatments are on the horizon and families need to be heard to advocate for ways to access them quickly.

Today, more than ever, it’s time to share their stories.

 

Join the fight!

Duchenne muscular dystrophy (DMD) is a rare disease with no cure. Orphan drugs that target its causes are being developed now. But we need your help. Research is the only hope. And research needs funding.For the past 20 years, Canadian families in the DMD community have been raising funds for and awareness of DMD. Their actions and successes have funded current research. Now that new treatments are emerging, families need to unite and be heard in order to access new treatments quickly.

Our part is to tell their story. Sharing these stories is one step forward towards the cure.

 

MAKE SURE NOT TO MISS OUR SHORT DOCS SERIES

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