Muscular Dystrophy Canada is thrilled to announce that after three years of working with each Canadian province and territory, all babies born in Canada can now be tested for spinal muscular atrophy (SMA). This milestone means infants diagnosed with SMA, the most fatal genetic disorder in children under two years of age, can receive life-changing treatment before symptoms even develop. It also marks the first neuromuscular condition to be added to screening panels across the country.
“Early diagnosis and effective treatment are critical to achieving the best possible outcomes for babies born with this neuromuscular condition,” said Dr. Pranesh Chakraborty , Chief of the Department of Pediatrics at Children’s Hospital of Eastern Ontario and Chair of the Department of Pediatrics at uOttawa’s Faculty of Medicine. “Historically, most infants and children with SMA would have been diagnosed only after they have developed weakness and respiratory difficulty, at a time when most of their motor neurons have been irretrievably lost. Now, instead of facing life-limiting disability and, in the most severe cases, a life expectancy of less than two years, babies affected by SMA every year in Canada shall be diagnosed within the first weeks of life allowing them to rapidly receive therapy and improved outcomes.”
The addition of SMA to newborn screening panels in all provinces and territories breaks down barriers and inequities families face simply because of where they live. It also acknowledges that screening for rare genetic diseases and access to early care result in positive health outcomes and long-term cost benefits for everyone. This is a tremendous accomplishment to start improving early detection and prevention.
We are so grateful for the partnership with Novartis Pharmaceuticals Canada, who helped us make this a reality across Canada. And a very special thank you to our dedicated community, generous donors and sponsors, Fire Fighter partners, clinicians, clients, and advocates for your unwavering support. We have helped make a life-changing impact on newborns and their families nationwide. We did it together—thank you!
For more information on this and other advocacy initiatives, please reach out through the research Hotline at 1-800-567-2873 ext. 114 or via email at research@muscle.ca.
Was your child diagnosed with SMA through newborn screening?
If so, we offer a specialized program called SMArTrack to help monitor, assess, and provide answers during the first two years of their life. For more information, or to get involved, please reach out to research@muscle.ca.
The 2024 Walk & Roll for Muscular Dystrophy Canada was a huge success thanks to you!
The Walk & Roll for Muscular Dystrophy Canada 2024 season is officially over, and it was a huge success, raising more than $1,089,000! Through 30 in-person events, we hosted more than 2,200 registered participants who joined us for fun-filled days, that helped break down the barriers faced by our neuromuscular community.
These special community events would not have been possible without the support of dedicated volunteers. We are so grateful to the 225 dedicated volunteers from across the country who donated their talent, skills and time totalling 1,000 volunteer hours to help make these amazing events the best they could be. Their hard work and enthusiasm helped create amazing events, and we are deeply thankful for their unwavering support and commitment to making a difference. Thank you!
This year, through a new initiative, we were thrilled to also support many individuals, groups, and schools with hosting their own Walk & Roll events in their local communities. These dedicated community members turned their commitment to breaking down barriers for the neuromuscular community into positive change by organizing, promoting and hosting their own Walk & Roll events. Thank you for your dedication and great work!
Thank you to all participants, donors, sponsors, volunteers, and everyone who participated, fundraised or attended a Walk & Roll for Muscular Dystrophy Canada event this year. Because of your hard work and dedication, Canadians affected by a neuromuscular disorder can continue to count on Muscular Dystrophy Canada for supportive programs and services, while we continue to fund ground-breaking research and champion advocacy efforts on their behalf. Thank you for making a difference and we hope to see you all again at next year’s Walk & Roll!
For more information on the Walk & Roll for Muscular Dystrophy Canada, visit walkrollmdc.ca.
Donor support makes a difference
When Sally and Cy Elston were approached by their longtime friend, Lloyd Lawrence, about supporting Muscular Dystrophy Canada, they rose to the challenge. Initially supporting a Dragon Boat Race fundraiser in Barrie, Ontario, their support has evolved over many years.
Now, Sally has continued the tradition they began together through her generous gifts to Muscular Dystrophy Canada. When asked why, Sally is clear. “It’s about people”, she says. “And the joy of knowing you are doing something for others”. While Cy passed away 15 years ago, Sally has remained steadfast in prioritizing philanthropic opportunities, “where (she) can do the most good for lovely people”.
Support from donors, like Sally, helps to eliminate the barriers that keep the neuromuscular community from reaching their full potential. Thank you for supporting our life-changing work. Every dollar matters and every donation is powerful!
Muscular Dystrophy Canada’s Annual Report is now available
2023-2024 marked another year of Muscular Dystrophy Canada “Breaking Down Barriers” for the Canadian neuromuscular community. If you would like to review Muscular Dystrophy Canada’s 2023-2024 Annual Report, please visit here.
Lise Poulin, Board Member and Stacey Lintern, CEO share highlights from Muscular Dystrophy Canada’s 2023–2024 Annual Report.
Clinical Trials in Canada for Neuromuscular Disorders Are on the Rise
In recent years, the number of clinical trials available in Canada for individuals with neuromuscular disorders has increased significantly, with opportunities doubling or even tripling in some cases. This is exciting news for people affected by neuromuscular disorders and their families as more trials mean greater access to emerging treatments, innovative research, and, potentially, life-changing therapies.
Clinical trials are research studies that explore new medical treatments, drugs, or therapies to determine their effectiveness and safety. These trials follow strict protocols and are essential in bringing new treatments to market. Clinical trials happen in phases, each with a distinct purpose. Early-phase trials (Phase 1) focus on safety and dosage, while later phases (Phase 2 and 3) explore the treatment’s effectiveness in larger groups. The more advanced the phase, the closer the trial is to delivering real-world treatments, with Phase 3 often being the final step before approval.
For individuals with neuromuscular disorders, participating in a clinical trial can offer access to cutting-edge treatments that are not yet widely available. The value of participating in a clinical trial extends beyond access to new treatments. Clinical trials contribute to medical knowledge that can lead to breakthroughs and better outcomes for future generations. By participating, individuals play an active role in their healthcare and help advance research that could lead to the development of a cure.
However, participating in clinical trials requires courage, grit, and a willingness to contribute to the scientific community. It often involves uncertainty, dedication, and perseverance, but it also means becoming a part of the journey to uncover new therapies and treatments that could one day transform care for all affected by neuromuscular disorders.
Navigating Clinical Trials: It’s Not Always Straightforward
While more trials are now available, navigating the clinical trial landscape can be complex. In some cases, like with Duchenne muscular dystrophy, there may be multiple trials available at the same time, each testing different treatments or approaches. Deciding which trial is right for you or your loved one can be challenging. Factors like trial eligibility criteria, location, and the type of intervention being tested must all be considered.
Muscular Dystrophy Canada Can Help
At Muscular Dystrophy Canada, we understand the importance of making informed decisions when it comes to clinical trials. We are here to help by sharing the latest clinical trials for various neuromuscular disorders and can provide timely information on available trials. We can help you find trials that match your specific condition, location, and eligibility criteria. Muscular Dystrophy Canada can assist in reaching out to trial coordinators and investigators to get answers to important questions about trial details, participation requirements, and more. We can also support families in interpreting clinical trial data, helping to make well-informed decisions.
If you’re curious about clinical trials and want to explore whether one might be right for you or your loved one, please contact research@muscle.ca.
Opening Doors: Navigating University with Neuromuscular Conditions
Choosing the right university or college is always a big decision, filled with considerations about programs, campus life, and future goals. But for students living with neuromuscular conditions, the decision becomes even more complex, as accessibility and support services are crucial to their academic success and well-being. The problem? There isn’t a centralized place where students can easily find information about the services, supports, and accommodations available at post-secondary institutions across Canada.
To address this gap, Muscular Dystrophy Canada is collecting data on a wide range of accessibility aspects, including physical spaces such as accessible classrooms and building, note-taking services and support for class materials, class structure and accommodations tailored to individual needs, residence options that cater to accessibility and personal care needs, transportation availability to and from campus, testing and exam accommodations for fair assessment, and washroom facilities and other essential amenities for universities and colleges across Canada.
What We Heard
In addition to gathering this data, Muscular Dystrophy Canada connected with students living with neuromuscular disorders who are currently enrolled in post-secondary institutions. We learned that:
- Many students are restricted in their selection of institutions due to their medical and accessibility needs.
- While some accommodations, like note-taking services meet basic needs, many students have additional unmet needs that negatively impact their academic performance and overall success.
- Inadequate accommodations provided by universities and colleges force students to pay out-of-pocket for extra support, adding a significant financial burden.
- Students with neuromuscular conditions often find there is limited financial assistance specifically tailored to their unique circumstances.
- Many university and college accessibility services lack sufficient knowledge about neuromuscular conditions, which limits the support available.
- Attendant care services are a critical and valuable resource that significantly enhances the experience of students with neuromuscular conditions. These services provide essential daily living support.
If you or someone you know is attending a post-secondary institution while managing a neuromuscular condition, Muscular Dystrophy Canada is here to help. We are dedicated to connecting you with the right resources and providing the information and support you need to reach your academic goals. Plus, thanks to the generosity of our donors, funds may be available to support post-secondary students—reach out to research@muscle.ca to learn more!
Help families, like Danielle’s, get the answers and support they so desperately need.
Danielle has defied all odds throughout her life. As a world-record holding Paralympian, Danielle challenged herself to win a medal, and not only did she win one, but over the course of two Olympic games, she won seven. Danielle is a published book author and engages many as a motivational speaker at events nationwide. She’s a proud mom of five and has earned an honourary doctorate. Danielle also has spinal muscular atrophy (SMA).
When she was two years old, Danielle was diagnosed and her lifelong connection to Muscular Dystrophy Canada began.
Because of our donors, Muscular Dystrophy Canada has been able to stand shoulder to shoulder with Danielle and her family throughout every stage of her neuromuscular journey, from supporting them as they navigated through some difficult questions, helping to provide vital equipment and working together to ensure the systems around Danielle were set up to ensure her success.
Please make a gift today and help families, like Danielle’s, get the answers and support they so desperately need.
“Not only did Muscular Dystrophy Canada provide emotional support to my family, but throughout my childhood they also provided support with equipment, advocacy, and navigating life with a neuromuscular disorder,” Danielle says. “Growing up, I had many questions: Would I be able to have kids? Would I pass this disorder on to my children? What would my prognosis look like? No matter what question or fear I had, I was always able to turn to Muscular Dystrophy Canada.”
Today, Danielle is thriving. But life isn’t always easy. Because receiving a neuromuscular diagnosis can be complex, Danielle received a new diagnosis recently. The good news is that this was possible because of our donors. Your investments ensured research continued to accelerate and change lives.
“Thanks to Muscular Dystrophy Canada’s donors, I have been able to finally get my official diagnosis of spinal muscular atrophy – lower extremities dominant (SMA-LED), after 10 years of genetic testing” Danielle says. “Thanks to this investment, I was also able to learn that my condition is genetic and there was a 50/50 chance it would be passed onto my children.”
Originally told she wouldn’t pass on her disorder, Danielle’s middle son, Samson was recently diagnosed with spinal muscular atrophy, just like his mom.
The path ahead for Danielle and Samson isn’t a straight line but is made easier because of Muscular Dystrophy Canada’s support, programs and services.
Our team is at the end of each phone call. We will answer every email, and we will help ensure Danielle, her family and everyone living with a neuromuscular disorder has every opportunity to meet their own goals, whatever they may be.
100 percent of Canada is now screening infants for spinal muscular atrophy, a potentially fatal disorder
Muscular Dystrophy Canada and partners deliver on goal outlined in Canada’s Rare Disease Strategy
Toronto, Ontario, Canada, August 20, 2024 – Muscular Dystrophy Canada is thrilled to announce that after three years of working with each Canadian province and territory, all babies born in Canada can now be tested for spinal muscular atrophy (SMA). This milestone means infants diagnosed with SMA, the most fatal genetic disorder in children under two years of age, can receive life-changing treatment before symptoms even develop. It also marks the first neuromuscular condition to be added to screening panels across the country.
“Early diagnosis and effective treatment are critical to achieving the best possible outcomes for babies born with this neuromuscular condition,” said Dr. Pranesh Chakraborty Chief of the Department of Pediatrics at Children’s Hospital of Eastern Ontario and Chair of the Department of Pediatrics at uOttawa’s Faculty of Medicine. “Historically, most infants and children with SMA would have been diagnosed only after they have developed weakness and respiratory difficulty, at a time when most of their motor neurons have been irretrievably lost. Now, instead of facing life-limiting disability and, in the most severe cases, a life expectancy of less than two years, babies affected by SMA every year in Canada shall be diagnosed within the first weeks of life allowing them to rapidly receive therapy and improved outcomes.”
In 2020, only Ontario and the Baffin region in Nunavut screened for SMA at birth. Today, regardless of where in Canada a child is born, they will receive the same screening; and, if SMA is diagnosed, the same healthcare, treatment and opportunity to thrive.
“I could not imagine what our life would look like if my daughter were not given genetic, SMA-testing at birth,” said Taylor Diakew, mother of a 2-year-old with SMA. “Today, thanks to her early diagnosis, and quick access to treatment, she is a happy, healthy little girl who does not exhibit any signs of SMA – she is walking, running, climbing, and talking like any child her age. Thanks to SMA newborn screening, she can live the best life possible.”
The addition of SMA to newborn screening panels in all provinces and territories breaks down barriers and inequities families face simply because of where they live. It also acknowledges that screening for rare genetic diseases and access to early care results in positive health outcomes and long-term cost benefits for everyone.
“It is a tremendous accomplishment to start improving early detection and prevention, one of the goals outlined in Canada’s Rare Disease Strategy. However, this success was only possible because of the willingness of provincial and territorial governments to work alongside Muscular Dystrophy Canada to add SMA to newborn screening. This is a significant step forward, and we hope it leads to the inclusion of other neuromuscular conditions on screening panels,” said Stacey Lintern, CEO, Muscular Dystrophy Canada. “We are grateful for the partnership with Novartis Pharmaceuticals Canada who helped us make this a reality across Canada and for every clinician, researcher, provincial laboratory lead, volunteer, donor, Canadian Fire Fighter, like minded organization, and government member who supported this project.”
“This initiative holds immense value for the entire Canadian neuromuscular and rare disease community, laying the groundwork for future transformative and life-changing initiatives,” said Dr. Homira Osman, VP of Research and Public Policy, Muscular Dystrophy Canada. “There are many progressive neuromuscular disorders where time is of the essence: early diagnosis and prompt access to treatments are critical drivers to achieving the best possible outcomes. Muscular Dystrophy Canada will now leverage the knowledge and findings gained from this initiative to ensure other neuromuscular disorders are included in newborn screening programs nationwide.”
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ABOUT MUSCULAR DYSTROPHY CANADA
Muscular Dystrophy Canada’s mission is to enhance the lives of those affected with neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for a cure through well-funded research. To learn more about Muscular Dystrophy Canada, please explore our website or call our toll-free number at 1-800-567-2873.
FOR MORE INFORMATION CONTACT:
Homira OsmanVice-President Research & Public Policy
Muscular Dystrophy Canada
Homira.Osman@muscle.ca
437-912-9037
YOU’RE MOVING RESEARCH FORWARD
Muscular Dystrophy Canada is the only dedicated source of funding for neuromuscular research in Canada. Because of donors, volunteers, Fire Fighters and incredible supporters, like you, we invested $900,000 into nine clinical and translational research projects through the Neuromuscular Research Grants Competition this year. Thanks to your generosity, we also invested $63,630 towards an innovative study led by Dr Gonorazky on Charcot-Marie-Tooth disease, bringing our research investment for this year closer to $1 million.
In recent years, neuromuscular research has undergone significant transformations, leading to changes in clinical standards, improved diagnoses and breakthroughs in treatment discovery. Continued investment in research is key to keeping and accelerating this momentum moving forward.
“We want to thank the clinicians and researchers on our Scientific Review Panel, as well our Lived Experience Readers who volunteered their time and expertise in selecting this year’s recipients.”
— Stacey Lintern, CEO,
Muscular Dystrophy Canada.
UNWAVERING COMMITMENT: FIRE FIGHTERS AND MUSCULAR DYSTROPHY CANADA CELEBRATE 70TH ANNIVERSARY
Through boot drives and tolls on local roads, community barbeques,
rooftop campouts, car washes, stair climbs and raffles – you name it – more than 600 fire departments, locals and associations across our country work with Muscular Dystrophy Canada every year so they can give all neuromuscular disorders “the boot” in this lifetime.
Fire Fighters have hosted these fundraising events for 70 years to support people affected by neuromuscular disorders.
“When it’s Fire Fighters and MDC together, we’re a family, and we know they need our help. So, when it comes to fundraising, I say, give me a challenge, and I’ll give you my best,” said volunteer Fire Fighter with Middlesex County, Launie Fletcher. Launie has been fundraising with Muscular Dystrophy Canada for 40 years!
Fundraising events also provide Fire Fighters with an opportunity to meet Canadians affected by neuromuscular disorders and see how they’re helping transform lives.
City of Edmonton career Fire Fighter, Courtney Polson added, “Events are just so rewarding for everybody because we see the difference they make. With Muscular Dystrophy Canada, I’ve seen firsthand where the money goes – how it makes a difference in people’s lives.”
To learn more about our 70-year partnership with Fire Fighters or to make a gift of appreciation, visit FilltheBoot.ca.
1954
by Fire Fighters over
70 years:
$103,607,542
$2.5 million
$3.2 million