News

2022-2023 YEAR IN REVIEW: ANNUAL REPORT HIGHLIGHTS

Accomplishments you made possible last year!

At its annual general meeting, the Muscular Dystrophy Canada Board of Directors officially issued its 2022-2023 Annual Report and what’s abundantly clear, is that thanks to your support we accomplished so much and broke down even more barriers for the neuromuscular community. Thank you!

  • We invested $2 million to fund: nine new clinical and translational science research projects through our Neuromuscular Research Grant Competition; six fellowships; two collagen 6-related dystrophy (Col6-RD) research projects in partnership with CureCMD; and, other projects to ensure the momentum we are seeing in research continues.
  • We partnered with Independent Living Canada, People First Canada, Inclusion Canada, Eviance, the Canadian Association of the Deaf, and the Canadian Council on Rehabilitation and Work to produce Shaping Caada’s Disability Inclusion Action Plan (DIAP), a report that will inform the federal government’s national Disability Inclusion Action Plan.
  • 491 individuals affected by neuromuscular disorders registered with MDC, an increase of 17% over the previous year. To accommodate this growth and provide support, we increased the funds directed towards Mission activities by 11%.
  • We diversified our fundraising by meeting new donors and better understanding their interests, and ensured all donors were being stewarded in ways that were meaningful for them. As a result, MDC increased its total fundraising by $2.6 million over the previous year for a total of $10.7M in revenue.

All of this was possible, because of people like you! Thank you for your commitment to MDC’s mission. We’re breaking down barriers for the neuromuscular community, together, so that sometime soon a breakthrough is made that leads to cures.

BOARD OF DIRECTORS

“I’m excited to welcome Fanny Chagnon, Liz Stirling, Lise Poulin, Nicole Cote and Michael Low to the MDC Board of Directors. Their wealth of knowledge, talents and familiarity with neuromuscular disorders will be invaluable for providing strategic guidance as we continue to break down barriers for the neuromuscular community this year. I am also extremely pleased that Debra Chiabai has stepped into the Chair position,” said Stacey Lintern, CEO, Muscular Dystrophy Canada.

New Board Chair Debra Chiabai added, “It has been a great pleasure to be a member of the Board as a Director for the last 13 years, and I am looking forward to stepping into a new role to help guide MDC through the next few years.”

Check out the entire report

QUEBEC JOINS THE RANKS OF PROVINCES SCREENING NEWBORNS FOR SMA

QUEBEC JOINS THE RANKS OF PROVINCES SCREENING NEWBORNS FOR SMA

We are thrilled to announce that spinal muscular atrophy (SMA) has been added to the newborn screening panel in Quebec, a significant milestone for the province and Muscular Dystrophy Canada (MDC). Screening is being integrated now, with full implementation to take place by the end of 2023.

Extensive conversations, collaboration and financial contributions ($583,778) towards the lab program at CHU de Québec-Université Laval, equipment, and building an evidence-based pathway for the early initiation of disease- modifying therapy for individuals with SMA across Quebec, led to this exciting change in policy.

“We couldn’t be happier with the recent news”, said Stacey Lintern, CEO, Muscular Dystrophy Canada. “The addition of SMA to the panel means that infants and families in Quebec will now equitably benefit from the same opportunities as the majority of other Canadians, gaining access to early detection possibly before the onset of symptoms.”

In neuromuscular disorders like SMA, early diagnosis and prompt access to treatments are critical to achieving the best possible outcomes.

“For families, what matters is for their child to have the best chance in life, to be able to do as much as they’re able to do. That is what we can offer with newborn screening for SMA”, said Dr Hugh McMillan, Professor of Pediatrics at the University of Ottawa, Pediatric Neurologist and Neuromuscular Specialist at the Children’s Hospital of Eastern Ontario (CHEO) and Clinical Investigator at the CHEO Research Institute. You can watch Dr McMillan’s video here.

We thank our partner Novartis Pharmaceuticals Canada Inc. as well as donors, clinicians, researchers, families affected by SMA, and Fire Fighters who collaborated with MDC to advocate for the inclusion of SMA on the newborn screening panel.

MDC is currently working with the Maritime provinces to bring newborn screening for SMA to that part of the country. For more information on MDC’s efforts in ensuring all Canadian babies are screened for SMA, visit muscle.ca/services-support/newborn-screening/

If you or a loved one was diagnosed with SMA, MDC has a variety of programs and services for you. Please contact us at 1-800-567-2873 or email info@muscle.ca for personalized support.

RESEARCH TRAINING IS AVAILABLE FOR YOU!

Did you know you could guide research and maximize its findings? It’s true! When you become a patient research partner you will inform research findings and make a difference for the NMD community!
Many incredible NMD community members have generously participated in research initiatives as participants. But now, you can be part of the team and direct research from the inside!

Canadians affected by NMDs, their families, and advocates are often asked to take part in research, but they often feel unprepared to do so. To help you feel ready to become a research team member, Muscular Dystrophy Canada (MDC) in partnership with the Neuromuscular Disease Network for Canada (NMD4C) offers online, self-directed, free training modules through the imPORTND Patient- Oriented Research Training program.

With a ‘nothing about me, without me’ approach, MDC has been working with parent and patient partners on different projects. Most recently MDC, together with researchers from McMaster University, and research teams from Germany and Italy, were selected to conduct collaborative research project looking at the transition experiences from pediatrics to adult care for people with Duchenne muscular dystrophy (DMD) and their families. This project team includes incredible partners with lived experience such as: Doyle Steinke, Nicola Worsfold, Mark Liam Arouza Pai, Gurvir Singh Shergill, Stacy Theofilopoulos, and Andrea Cleary. Become an MDC research partner today, to participate in projects that will have a direct impact on the NMD community.

“As parents of adults with DMD and developmental disabilities, we have lived through a very frustrating transition regarding our boys’ care. From the medical side, the transition to adult care has been reasonable but from the side of caregiving and our role as caregivers, we are not being looked after like we were when the boys were minors. This is a gap that seems to plague the care system. Too many adults with complex needs fall through the cracks during the transition to adulthood. Being directly involved in this research project gives me the opportunity to make positive changes for others in similar situations.”

Doyle Victor Steinke, patient research partner

Interested in becoming a research partner? Contact us at info@muscle.ca, call 1-800-567-2873 or visit muscle.ca/services-support/advocacy/importnd/ to learn more!

MYASTHENIA GRAVIS JOURNEY MAPPING: AN INSIDE LOOK

In 2023, Muscular Dystrophy Canada (MDC) conducted a journey mapping initiative for Canadians with myasthenia gravis (MG), a rare autoimmune neuromuscular condition. While some treatments are available for MG in Canada, there remains many challenges and unmet needs. We recognized these recurring roadblocks and difficulties in the diagnosis process for our MG clients, and wanted to capture this information so we would know how best to support this community.

We sought to examine the journey from time of most bothersome symptoms, to diagnosis, to treatment, to post-diagnosis. So, we asked individuals affected across Canada to participate in surveys, interviews and roundtable discussions related to their experiences with their diagnosis, treatment, information provided and day-to-day life.

Our goal was to capture the clinical, attitudinal, cost and informational journey including time to diagnosis, diagnostic experience, treatment, emotions during each stage of their journey, and pressure points. The MG journey map below illustrates a person’s needs, processes they follow, and perceptions and emotions they have throughout their healthcare journey. Information is key, knowing where the challenges are means we know exactly where we need to support our community!

Want to learn more about the MG Journey Mapping project?

Visit: muscle.ca/services-support/advocacy/journey-mapping/ to watch the results video and see the MG Journey map.

Important Update on Phone Solicitations

Muscular Dystrophy Canada does not use telemarketing services. You can support Fire Fighters and help them Fill the Boot for Muscular Dystrophy Canada and the individuals and families affected by neuromuscular disorders by going to filltheboot.ca.

STATEMENT REGARDING PHONE SOLICITATIONS

Several years ago, the Canadian Fire Fighter Curling Association (CFFCA) retained a telemarketing company to assist in fundraising for their annual curling event. A portion of the funds raised in past years were donated to MDC.

Muscular Dystrophy Canada can confirm a campaign soliciting donations for the Canadian Fire Fighter Curling Association has begun, seeking donations that support the Canadian Fire Fighter Curling Association.

In the past, we received complaints regarding the tone and approach of these solicitation calls and brought this to the attention of the CFFCA President.

Should you have any concerns or questions please contact the Canadian Fire Fighter Curling Association directly at cffca.ca.

We sincerely thank all of our generous supporters for making our work possible. Again, to help Fire Fighters #filltheboot go to filltheboot.ca.

Governments across Canada urged to close current gap in care for debilitating neuromuscular disease by funding treatment advancements

Toronto, Ontario – Access to innovative care is vital for Canadians living with myasthenia gravis (MG) to help relieve the considerable impact faced by those affected by this rare neuromuscular disorder. For the first time in over 30 years, treatment advancements for adults with refractory MG have been approved in Canada, bringing hope to those for whom current treatments are ineffective. Yet, none of these new treatments are accessible through public funding, including Soliris (eculizumab), which was the first to be approved in 2018.

“Today, Canadian patients affected by MG who rely on public funding unfortunately spend years navigating challenging processes to show the inefficacy of treatments, only to find out they have run out of accessible options,” says Stacey Lintern, CEO, Muscular Dystrophy Canada. “We are urging provincial and territorial governments to help close the gap in care for Canadians living with myasthenia gravis by immediately enabling access to therapeutic advancements in MG.”

Myasthenia gravis (MG) is a severely debilitating chronic condition that causes progressive muscle weakness leading to an inability to swallow, decreased mobility, respiratory crises, and hospitalization. The diagnosis of MG is often missed or delayed and can take five years, on average, to obtain. The fluctuating and unpredictable nature of MG symptoms, with periods of worsening and remission, has a substantial impact on quality of life. Thus, the main goal of treatment is to achieve either long-term remission or reduce the severity of disease to mild or minimal symptoms.1

“The extensive hospital visits and critical care resources required to manage an MG crisis can impact the physical, emotional, social and financial well-being of a person, but it also places a financial and resource burden on the provincial healthcare systems,” says Chloe Atkins, who is living with MG. “Earlier public funding of innovative treatments which more effectively control the disease could result in fewer hospital admissions and less damage to the body, meaning a greater quality of life for people living with MG and a decreased strain on our healthcare services.”

In response to the persistent gap in care, the MG community, including medical experts from across Canada, have come forward to express the importance of having immediate access to innovative treatment options for the disease.

“Patients with MG need access to more and different strategies to address both the burden of the disease and the burden of existing therapies,” explains Dr. Hans Katzberg, a neurologist and a member of NMD4C – The Neuromuscular Disease Network for Canada. “Newer treatments for refractory MG and moderate to severe non-refractory MG have the potential to improve the patient’s quality of life by decreasing the frequency and intensity of symptoms, and by helping to reduce the dosage and usage of other medications with higher toxicity or delayed onset of action.”

Following the approval of Soliris, two other drugs for the treatment of MG were approved in Canada – Ultomiris (ravulizumab) in January 2023 and Vyvgart (efgartigimod alfa) in September 2023. However, none of these treatments are currently accessible to patients in Canada through public funding.

“Patients living with MG in Canada should not need to wait for access to more effective, better-tolerated treatment innovations,” underscores Ms. Lintern. “There is an immediate pathway to providing public funding for these treatments across the country, which will help relieve the significant treatment burden that currently impacts those living with this debilitating disorder.”

Muscular Dystrophy Canada appeals to the provincial and territorial drug plans to expedite publicly funded access to all Health Canada-approved treatment options for myasthenia gravis, now and in the future.

– 30 –

References:
  1. Mapping the Myasthenia Gravis Canadian Journey – Muscular Dystrophy Canada, 2023

ABOUT MUSCULAR DYSTROPHY CANADA

Muscular Dystrophy Canada’s mission is to enhance the lives of persons affected by neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for cures through well-funded research. To learn more about MDC, please visit muscle.ca or call our toll-free number at 1-800-567-2873.

FOR MORE INFORMATION CONTACT:

Heather Rice
Muscular Dystrophy Canada
Heather.Rice@muscle.ca
902-440-3714

Mark got what he needed to get back outside and explore nature!

“My son is a 14 year old boy with DMD who was diagnosed when he was just one and half. Despite the physical limitations, Mark loves nature and outdoor time. But, we have to closely monitor the steps he takes so that he is not overexerting himself which will result in muscle cramps or even permanent damage. So sadly, we had to press pause on some outdoor activities until we could get him a manual wheelchair. It was not easy to find a good sized wheelchair for him to use safely outside. The health insurance only covers some portion of the cost for the customized chair we got for him. MDC reached out the helping hand as soon as they got to know about our needs and provided incredible support mentally, emotionally and financially. Thanks to MDC, our son can now go out and enjoy the fresh air of the forest with more mobility and independence. Life doesn’t have to be perfect to be wonderful. MDC’s life-changing support always comes at the perfect time and makes our lives better. We know there are so many heroes with capes working very hard to support us and other families in need.”

Mandy Liu, ON

Community Statement: Update on Deflazacort®/Calcort® Availability

We want to bring your attention to recent developments surrounding the availability of Deflazacort®/Calcort®, a corticosteroid vital to many Canadians affected by Duchenne muscular dystrophy. Up until 2023, Deflazacort was accessible at a relatively low cost in Canada through Health Canada’s Special Access Program despite intermittent supply shortages. In February, due to a change in the manufacturer, the price of Deflazacort was increased by a staggering 400%. The price increase led to an initial disruption in supply and access but was fully resolved by spring.

In July, the Canadian distributor notified us about delays in the production of Deflazacort originating from issues at the manufacturer’s end. Despite our ongoing attempts to seek updates and influence change, the supply challenges persisted, directly impacting individuals with Duchenne and their families throughout Canada. The positive update is that we received communication last week from the manufacturer, Cheplapharm, indicating that the supply is set to be restored and will soon be accessible again in Canada. The supply of Deflazacort for Canada is undergoing quality control procedures in their warehouse and they have assured us that they will prioritize the delivery to expedite the availability of Deflazacort in Canada. If you are experiencing challenges accessing Deflazacort®/Calcort®, or your pharmacy has questions about this product, please contact us at research@muscle.ca or call 1-800-567-2873 ext. 1114.

We should note that in light of the recurring issues related to supply, stock shortages, and pricing, MDC is eager to collaborate with you, our valued community members, to navigate the optimal approach to addressing these changes. Together, we remain committed to advocating for improved access, affordability, and timely availability of essential medications and therapies for the neuromuscular community.

Thank you,

Stacey Lintern
Chief Executive Officer
Muscular Dystrophy Canada

Your support has improved outcomes for thousands of Canadians, like Ben

“We thought our child was going to die a very slow and painful death. Our world ended, and we were never going back to life as we knew it.” Those were Amanda and Brodie’s first thoughts when they learned their son, Ben, was affected by Duchenne muscular dystrophy (DMD).

For the first three years of Ben’s life, countless doctors and physiotherapists told his parents that he suffered from low muscle tone. Unable to crawl, or walk unassisted, Ben was referred for blood tests. “When the doctor told me he had muscular dystrophy, she sent me information that told me I should expect my son to be in a wheelchair by age six, on a ventilator by age eight and dead by 19,” Amanda says. “It was horrific, devastating news.”

When a Canadian is diagnosed with a neuromuscular disorder, Muscular Dystrophy Canada (MDC) knows they will need a lot of support and have many questions. Your gift today will give families hope. When you’re facing a life-changing diagnosis for your child or family member, hope can make a huge difference.

Donate now!

Since our inception, MDC has been a leader in funding ground-breaking research for all neuromuscular disorders. For decades, MDC has been helping to accelerate the development of more treatments, therapies, and hopefully, one day soon, potential cures. It is an exciting time for neuromuscular research and we are proud to see incredible advancements that are having real-world impacts on families like Amanda and Brodie’s.

After the initial shock of Ben’s diagnosis wore off, and Amanda and Brodie were able to digest the information they had received, they were referred to MDC by their healthcare provider. Armed with new information about therapies and medication available, Brodie and Amanda started to have hope once again. “I remember feeling that finally there was a light at the end of the tunnel”.

By donating today, you are investing in families like Brodie and Amanda’s. Whether through research investments or helping them navigate upcoming treatments for Ben, your gift can enable so much. Thanks to you and others like you, we can continue to invest in Canadians affected by neuromuscular disorders in the ways that matter most to them.

“The funding that MDC has provided to neuromuscular research has changed our lives infinitely,” Brodie says. “We have seen directly, even from the information we received initially, how far medicine and therapies have come in as little as 10 years. Ben has great doctors who are pushing the envelope to find the next thing – a treatment, therapy or cure. The funding from MDC specifically enables them to do that research and take those chances.”

Being diagnosed with a neuromuscular disorder is very difficult and often shocking. Your mind races between practical concerns and anxious thoughts: what treatments are available? What will happen to me in the next five or 10 years? Who can give me the answers I can trust? By donating to MDC today, you are ensuring families have the answers they desperately require, ensuring hope in the months and years to come. You can help provide clarity and comfort to a family during that difficult time.

Your gift today will ensure that families have access to the most up-to-date information they need when they need it most.

Thanks to improvements in neuromuscular treatments and therapies, today, Ben is an active six-year-old who is able to walk, run and climb. And while Amanda and Brodie are still concerned with what the future holds, they know that Duchenne is a very different disease now, compared to 20 years ago. “Ben has a very serious disease and kids still die from it, but Duchenne is not the disease it used to be. DMD comes in all shapes and sizes, which is thanks, in large part, to how far medicine has come. It is a heavy load to carry, but it would be a lot heavier without the support of MDC.”

Your generosity today will help support MDC’s programs and services, while also investing in critical neuromuscular research. Together, we can provide hope, change the face of muscular dystrophy and other neuromuscular disorders, and improve the outcomes now and for future generations. But we cannot continue this important work without your support.

“Understanding the support that MDC provides to Canadians is huge,” Brodie says. “There is a group of people – the MDC village, that are working day and night on behalf of my family. It affects our day-to-day life and I only see some of it – there is so much work happening beyond the support they provide to our family.”

Please consider making a gift today that will help countless Canadians right now. You can change the future and give hope to future generations.

Donate now!