2020 Year in Review

While we look ahead with hope, possibility, and vision, we take time to reflect on a year like no other.

We started January 2020 with fresh excitement to celebrate our 25th anniversary and share a new three-year strategic plan focussed on engaging Duchenne families across Canada like never before.

We are proud of what we accomplished through unprecedented challenges, made possible by YOU – our donors, volunteers, and supporters.

Join us in reflecting on our achievements and inspiring hope for the year to come by reading Defeat Duchenne Canada 2020 Year in Review below.

Through your support, we have seen significant advancements in the quality and longevity of life for boys and young men with Duchenne. Yet while we celebrate the progress made, we recognize that we are still in a race against time to find a cure and a fight to ensure Canadians have equal access to treatments once they are discovered.

From everyone at Defeat Duchenne Canada, thank you for your continuous support in the mission to defeat Duchenne.

Help inspire others and raise awareness of Duchenne by sharing this link through email and social media: https://bit.ly/38ro9dd

The 2020 Year in Review can also be found on all of our social media channels – click the buttons below to share, like, and comment and help spread the message far and wide.

2021: New Year Filled with Hope

We’re starting 2021 filled with gratitude. Throughout November and December, you picked up the pieces to the complex puzzle of Duchenne muscular dystrophy and helped create the beautiful mosaic of HOPE.

With your generosity, we surpassed our goal of $100,000 to invest in research that will allow boys and young men around the world to live longer, healthier lives.

This would not be possible without the vision of seven families who cumulatively donated $50,000 to match each gift.

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Thank you for helping put a stop to this life-limiting disease.

P.S. Click the button below to learn more about the campaign and the Prendergast family.

Defeat Duchenne Canada: Brand Awareness Survey

As Defeat Duchenne Canada moves forward with future marketing and communication initiatives, we want to know:

What does the Defeat Duchenne Canada “brand” mean to you?

We ask that you share your thoughts by completing a short survey – only about 10 minutes – which will help provide us with insights and direction that will enhance our marketing and communication effectiveness.

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If the button does not work, try clicking the link here: https://bit.ly/3aCRgtA.

Please Note:

  • If there are others in your household connected to Defeat Duchenne Canada or Duchenne muscular dystrophy, we encourage them to complete the survey from their perspective as well. If they are using the same computer, please ensure each individual starts and submits the survey before the next person begins.
  • It’s best completed on a desktop computer or tablet because of their larger screen sizes.
  • The survey is entirely anonymous.

Please complete the survey in one sitting by the end of the day, Wednesday, September 2, 2020 (the survey may time out if you leave and come back later).

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A generous donor will contribute $1 for each individual who completes the survey to a maximum of $1,000.

“We are proud to donate up to $1,000 for this survey participation.”

“Our family is delighted to sponsor this research study, and we would like to thank you for taking the time to fill out your response. We know that this investment into Defeat Duchenne Canada is going to pay huge long-term dividends to the organization, and most importantly, to the families the organization supports.

Often people like to donate to research or other more common areas, but in our experience, it is operational items like this survey that give the best return on our investment for our donation. We are grateful to the organization to let us anonymously support this opportunity.”

– R & B

Only one submission per person will be counted.

If the button does not work, try clicking the link here: https://bit.ly/3aCRgtA.

If you have any questions at all, please contact Manager of Marketing and Stakeholder Engagement, Rochelle ten Haaf, at rochelle@defeatduchenne.ca.

We appreciate your candid comments and responses. They will be very important as we chart our future and effectiveness as an organization to defeat Duchenne.

On behalf of the Duchenne community, we thank you.

Defeat Duchenne Canada + Muscular Dystrophy Canada (MDC): Return to School Webinar

Defeat Duchenne Canada and Muscular Dystrophy Canada (MDC) were very pleased to join forces to share information on returning to school for children with neuromuscular disorders.

Many parents, caregivers, and guardians face new and difficult choices about how their child will return to school in the fall, such as deciding between in-person, virtual learning and home school. This webinar is designed to help parents, caregivers, and guardians of children with neuromuscular disorders weigh the risks and benefits of available educational options to help them make decisions about sending their child back to school.

Return to School: Considerations for Children with Neuromuscular Disorders

Friday, August 21, 2020, | 12:00 PM EST

Panellists:

  • Dr. Ronald Cohn, President and CEO at the Hospital for Sick Children
  • Dr. Laura McAdam, Clinician Investigator, Pediatrician at Holland Bloorview Kids Rehabilitation Hospital
  • Angela Nardi-Addesa, System Superintendent – Leadership, Learning and School Improvement, Special Education and Inclusion at the Toronto District School Board
  • Janine Small, Centrally Assigned Principal at the Toronto District School Board
  • Nicola Worsfold, Director of Research and Advocacy at Defeat Duchenne Canada, Parent

Congratulations: Dr. Rudnicki Elected as Fellow of the Royal Society

Defeat Duchenne Canada would like to congratulate Dr. Michael Rudnicki, Chief Scientific Officer of Satellos Bioscience and a Defeat Duchenne Canada research funding recipient, for being elected as a Fellow of the Royal Society, the United Kingdom’s national academy of sciences.

The Royal Society is the world’s oldest and most esteemed scientific institution founded in 1660 and comprises the likes of Isaac Newton, Charles Darwin, and Albert Einstein. Dr. Rudnicki has explicitly been recognized for his fundamental discoveries in the identification and characterization of muscle stem cells and their role in Duchenne muscular dystrophy.

Read the full press release here.

To learn about the Duchenne research currently funded in collaboration with Muscular Dystrophy Canada (MDC), click here.

Defeat Duchenne Canada + Muscular Dystrophy Canada (MDC): Burden of Illness Study

Establishing the burden of illness for a rare disease, like Duchenne muscular dystrophy, is critical for Canada’s access to medicines.

Defeat Duchenne Canada has partially funded a burden of illness study currently underway by Muscular Dystrophy Canada (MDC), and we need your support! Please see the details below and complete the online survey before Monday, July 6, 2020:

About the Study

You are invited to participate in a web-based online survey on the direct and indirect costs associated with living with or caring for someone with a neuromuscular disorder and impacts on quality of life. This is a research project being conducted by Muscular Dystrophy Canada.

The purpose of this survey is to collect individual – and caregiver – reported outcomes to better understand the burden of neuromuscular disorders on patients and their caregivers in Canada.

It should take approximately 1 hour to complete. If necessary, you can save and return to the survey at any time.

You can also email research@muscle.ca or call 1 800 567-2873 ext. 9037 to schedule a time to complete the questionnaire by phone.

Defeat Duchenne Canada: 2019/20 Research Grants Announced

Since 1995, Defeat Duchenne Canada has strongly believed in strong partnerships with academics and clinicians to fund the most promising research for Duchenne muscular dystrophy. This focus and fundraising effort have brought us to granting more than $13.1 million across 45 research projects.We have attracted applications from leading Duchenne researchers from all over the world including the United States, UK, Netherlands and Japan, and in 2020, we received the highest number of grant applications in our 25-year history!

Our vision is a world free from the physical and emotional anguish of Duchenne muscular dystrophy. We know we can get there, and we know that research is the key. But we also know this: if research doesn’t get funded, it doesn’t happen and if we don’t advocate for access to treatments once they are discovered, our boys and young men will suffer.We would not be here if it weren’t for the generosity and vision of our donors – thank you for your continued support to #defeatduchenne.

Click here to learn more about the 2019/20 research grant recipients and our continued research funding.

Defeat Duchenne Canada: The loss of an incredible volunteer, Ed Coxworthy

Defeat Duchenne Canada has lost one of its greatest supporters.

The man we nicknamed “The King of Newfoundland,” Ed Coxworthy, the motorhome driver during the cross Canada journey in 1998-99 passed away Sunday, June 14, 2020, at his home on Bell Island, Newfoundland. Ed was 80 years old.

He may have had a gruff exterior, but inside Ed had a heart of gold and would have done anything to help Defeat Duchenne Canada and all the boys with Duchenne muscular dystrophy.

It was probably a bit of luck mixed with divine intervention that brought Ed to Defeat Duchenne Canada (pictured here, far right). For a long time, he was in the background of the story, which began with a letter written in London and the message received on the ferry from Portugal Cove to Bell Island.

My friend, former broadcaster Peter Garland, who was born and grew up in Newfoundland had written to a magazine called the Downhomer, a good-news magazine with content that celebrates life, and all the good things that go with it. The magazine is a link to home and is mailed to Newfoundlanders all over the world.

There on page 57 was Peter’s letter urging Newfoundlanders to support Defeat Duchenne Canada and a determined dad who was about to begin his cross Canada journey at the little harbour at Quidi Vidi, on the edge of St. John’s, Newfoundland.

Ed’s wife Kay, who had just joined the staff at the Downhomer was reading the latest edition of the magazine as she took the ferry home to Bell Island from the mainland.

The next day Kay went into the office of the magazine’s editor and said to him, “We’ve got to help this man,” and the editor said, “You make it happen girl!” That set the wheels in motion.

For the next four months, Kay Coxworthy and Peter Garland’s sister Joanne Dawson, who lives in Summerford, Newfoundland, worked tirelessly by telephone, by letter and by fax (email was pretty much unheard of in those days), to find us places for to eat and sleep all across Newfoundland in those early days on the road.

It wasn’t until the night before our journey began that Ed’s wife Kay and Joanne finally met each other in person.

It was during a traditional screech-in, where come-from-aways—or CFA’s—are invited to recite an age old saying, eat a piece of bologna (also known as a Newfoundland steak), kiss a cod and then take a shot of Screech, a potent West Indies rum. If you were not born on the rock, then you become an honorary Newfie by being officially ‘screeched in.’

On their way to the screeching-in, Kay turned to her husband Ed and said, “You know they’re still looking for a driver for part of Newfoundland.” Ed, who had worked as an underwater welder, a lobster fisherman, a seal hunter, a transport truck driver and had served Canada overseas as a Peacekeeper in Cypress, said to Kay, “Don’t be getting me involved in any fundraisers. I’ve done my part for the country, thank you very much.”

On their way home, after listening to what I had to say about Jesse and boys with Duchenne and why I was about to try to walk across the country to raise awareness and dollars for research, Ed said to Kay, “Well, maybe I’ll go for a week.” Ten months later he went home.

For the 286 days we were on the road, Ed’s humour made tough days seem like fun. His one-liners were classics.

I remember a day in the Maritimes when Ed came back from the grocery store and he looked at me and said in his unique Newfoundland way of speaking, “By the Jesus Johnny, the deli in that grocery store, they cut that meat so thin—it only had one side.” And so it went, day after day with my friend Ed Coxworthy, the unsung hero I was happy to know for the next 22 years of his life.

He was listed as the motorhome driver, but he was also our cook and I will never forget the dark, soaking wet night we had our motorhome Thanksgiving dinner of ham, turnip, carrots, cabbage and yellow peas pudding, prepared by a man who six-months earlier had said, “Maybe I’ll go a week.” He probably never knew it was him I gave thanks for on that day.

The stories about Ed Coxworthy became part of the magic of Defeat Duchenne Canada. Grit, perseverance and a remarkable dedication were all in the gruff exterior that housed a heart of gold.

This is what being Canadian and taking action in caring for others looks like. I hope you can see a bit of yourself in Ed’s eyes and I hope you too will do all you can to make a difference in the lives of others—just the way my friend Ed Coxworthy did.

Thanks for everything, Ed. Rest in peace and I’ll see you somewhere down the road.

– John Davidson, Founder of Defeat Duchenne Canada

Defeat Duchenne Canada: $1M Grant Towards Clinical Trial

Defeat Duchenne Canada grants $1M toward the launch of a clinical trial investigating vamolorone for Duchenne muscular dystrophy in Canada.

Defeat Duchenne Canada, Canada’s leader in Duchenne muscular dystrophy funded research, is proud to announce a $1M grant towards a clinical trial evaluating vamorolone for the first time in children 2-4 years of age prior to the onset of Duchenne, and adolescents 7-18 years of age already living with Duchenne, the most common fatal genetic disease diagnosed in childhood.

This announcement marks two ground-breaking achievements for the Canadian charity – the first time granting one million dollars to a single project and directly funding a clinical trial for older children and adolescents, an age range often neglected in clinical trials.

Dr. Eric Hoffman and his team at ReveraGen BioPharma have found a way to tweak the chemistry of standard corticosteroids, the current standard of care, and developed a new molecule that retains the anti-inflammatory benefits but loses much of the activity associated with side effects.

ReveraGen has successfully completed Phase I and II programs demonstrating treatment with vamorolone led to improvements of strength and mobility over six months and preserved over a year-and-a-half of treatment. Importantly, key side effects, such as stunting of growth, were not observed, and the boys grew normally. This data is supportive of vamorolone having the potential to replace corticosteroid as the standard of care treatment for children and young adults with Duchenne.

This game-changing research would not be possible without the financial investment by Defeat Duchenne Canada’s.

“The VBP15-006 clinical trial funded by Defeat Duchenne Canada will provide the first vamorolone treatment data in very young treatment-naive boys with Duchenne (2 to 4 years old), as well as the first data in older boys with Duchenne already treated with corticosteroids and transitioned to vamorolone. Families living with Duchenne across Canada and the academic medical centers have been amazing collaborators with the vamorolone development team. We are very excited to be able to continue this relationship.”

Dr. Hoffman

The funding from Defeat Duchenne Canada ensures that sites across Canada will be supported to recruit boys with Duchenne into the new study.

“Defeat Duchenne Canada relies on the support of our donors, knowing that research is the road to hope for this devastating disease. Most children with Duchenne are using a wheelchair by the time they reach their early teens, and with the potential for new treatments powered by research, like this study, we hope to delay the progression of this disease.”

Nicola Worsfold, Director of Research and Advocacy at Defeat Duchenne Canada

“Our mandate as a Duchenne patient organization is to speed up the process of drug discovery and access to beneficial medications addressing all stages of the disease. The VBP15-006 clinical trial addresses the unmet need for inclusion for an expanded age range. We look forward with hope that this potential steroid alternative can increase the quality of life for children and young adults with Duchenne,” continues Nicola.

The $1M research grant is provided under a new “return-on-investment” or “venture philanthropy” model for Defeat Duchenne Canada, where the funding will be repaid to the organization based on later drug sales of vamorolone internationally. If vamorolone is successful, Defeat Duchenne Canada will receive over 400% return on its investment in vamorolone. The returns can then be used to fund further philanthropic efforts by the organization. Other international non-profit organizations, such as Duchenne UK and Muscular Dystrophy Association in the United States, have also partnered with ReveraGen for this project and other clinical trials of vamorolone.

“Our investment in this late-stage clinical development program holds great promise for our boys and may also provide a sustainable funding model for the future of the organization,” concludes Nicola.

https://youtu.be/wUGZU7Pc2s8

About Defeat Duchenne Canada

Defeat Duchenne Canada is Canada’s leading charity in the fight to defeat Duchenne muscular dystrophy – the most common fatal genetic disease diagnosed in childhood. For the past 25 years, Defeat Duchenne Canada has empowered patients, families, and caregivers living with Duchenne through education and resources, provided a collective voice to advocate for access to treatments in Canada, and has become the country’s largest funder of Duchenne research investing more than $13.1M in projects around the world.

About Duchenne muscular dystrophy

Affecting approximately 1 in every 5,000 boys in Canada, Duchenne gradually weakens the body’s muscles due to the loss of the protein, dystrophin, ultimately leading to the deterioration of vital organs, such as the heart and lungs. Most kids with Duchenne use a wheelchair by the time they are 12, and with improved care powered by research, more people with Duchenne are living into their early 30s. While we celebrate the progress made, we are still in a race against time to find a cure, and must fight to ensure Canadian boys and young men have equal access to treatments once discovered.

About ReveraGen BioPharma

ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGen’s lead compound, vamorolone, has been supported through partnerships with foundations worldwide, including Muscular Dystrophy Association USA, Parent Project Muscular Dystrophy, Foundation to Eradicate Duchenne, Save Our Sons, JoiningJack, Action Duchenne, CureDuchenne, Ryan’s Quest, Alex’s Wish, DuchenneUK, Pietro’s Fight, Michael’s Cause, Duchenne Research Fund, and Defeat Duchenne Canada. ReveraGen has also received generous support from the US Department of Defense CDMRP, National Institutes of Health (NCATS, NINDS, NIAMS), and European Commission (Horizons 2020).

www.reveragen.com

About vamorolone

Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors [1, 2]. This has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore could replace existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is significant unmet medical need in this patient group as high dose corticosteroids have severe systemic side effects that detract from patient quality of life. Phase 1 studies in adult volunteers [3], and Phase 2a studies in 48 DMD boys [4] showed biomarker studies consistent with a partial agonist mechanism of action, with dose-responsive improvements in both efficacy and safety biomarkers. Dose-finding studies with 24-weeks of vamorolone treatment over a dose range of 0.25 to 6.0 mg/kg/day showed dose-related improvements in multiple measures of muscle strength and endurance [5]. Vamorolone has been granted Orphan Drug status by both FDA and EMA, Fast Track designation by the FDA, and Priority Innovative Medicine designation by the UK MHRA.

[1]Heier CR at al. (2013). VBP15, a novel anti‐inflammatory and membrane‐stabilizer, improvesmuscular dystrophy without side effects. EMBO Mol Med 5: 1569–1585 [2]Heier CR et al. (2019). Vamorolone targets dual nuclear receptors to treat inflammation anddystrophic cardiomyopathy. Life Science Alliance DOI 10.26508/lsa.201800186 [3]Hoffman EP et al. (2018). Phase 1 trial of vamorolone, a first-in-class steroid, showsimprovements in side effects via biomarkers bridged to clinical outcomes. Steroids 134: 43-52. [4]Conklin LS et al. (2018). Phase IIa trial in Duchenne muscular dystrophy shows vamorolone isa first in-class dissociative steroidal anti-inflammatory drug. Pharmacological Research 136:140-150. [5]Hoffman EP et al. (2019). Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology 93: e1312-e1323

Defeat Duchenne Family Forum Montréal: New Dates!

Due to COVID-19, the Defeat Duchenne Family Forum presented by PTC Therapeutics and in partnership with La Force DMD has been transformed from a one-day “in-person” program into a series of four virtual events taking place May 20 – 30, 2020.

Join us as families and caregivers navigating the Duchenne journey across Canada come together with researchers, clinicians, and industry professionals to learn about the latest in Duchenne care and research.

Learn more and register today.