News

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.

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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!

Quebec joins the ranks of provinces screening newborns for life-threatening disorder

FOR IMMEDIATE RELEASE – Muscular Dystrophy Canada (MDC) is 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 MDC.

“Congratulations to the Government of Quebec on this step, which will lead to early diagnosis and treatments that will have life-changing results for individuals and families,” said Stacey Lintern, CEO, Muscular Dystrophy Canada.

Screening is being integrated now, with full implementation to take place by the end of 2023.

Over the past two years, MDC collaborated with Novartis Pharmaceuticals Canada Inc. as well as clinicians, researchers, people affected by SMA and families, Fire Fighters and donors to advocate for the inclusion of SMA on the newborn screening panel.

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 rapid early initiation of disease-modifying therapy for individuals with SMA across Quebec, led to this exciting change in policy.

“It was important for MDC to go beyond supporting the necessary infrastructure for SMA newborn screening, and to also ensure families receive the best quality care in the event of a positive screening result,” added Lintern. “We couldn’t be happier with the recent news. 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.”

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

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For more information, contact:

Sylvie St-Amand
Translation and Communication Specialist
Muscular Dystrophy Canada
Phone: 514-244-0381
Email: sylvie.st-amand@muscle.ca

Freya’s life has been changed, thanks to your amazing support

For many, the birth of a child is an exciting, and joyful event. Freya’s birth was no exception. Born last year, Freya was small, beautiful, and one of the first babies to be screened for spinal muscular atrophy (SMA) in Alberta.

In 2020, MDC began work to positively influence policy change, and get SMA included on all existing provincial newborn screening panels across the country. We’re nearly there, as today more than 75% of regions across Canada are offering this life-changing test. This is a huge step towards securing early diagnosis and access to treatment for the next generation of Canadians.

By making a gift today, you can help ensure Canadians of all ages have access to early diagnosis and treatment options.

Donate now!

“When Freya was a few days old, we were called in to get confirmation testing done on her, and her SMA diagnosis was confirmed when she was just two weeks old,” says Taylor, Freya’s mom. “The whole process was quite simple and quick. Once diagnosed, Freya was quickly approved for treatment which she received at 29 days old.”

One in 10,000 babies are born with SMA each year in Canada. Without early diagnosis and access to treatment, many infants will not live past the age of two. Thanks to your gifts, we are close to ensuring all babies across the country, like Freya, are screened and receive treatments that have life-changing results.

Today, Freya is an active toddler, exhibiting no signs of SMA. She is walking, running, climbing and talking like any other child her age.

Your gift today will enable MDC to continue advocating for improved diagnosis and treatment options, ensuring that all Canadians affected by a neuromuscular disorder, regardless of type, have access to the support and treatments that are right for them. Donate today to make a life-changing impact.

“Freya is a happy and healthy child, and I could not imagine what our life would look like if she were not given genetic, SMA-testing at birth,” Taylor says. “The challenges Freya and our family face because of her disorder are very minimal, thanks to early diagnosis and treatment. And while we have had many questions since her diagnosis, the support and information we have received from Freya’s doctors and MDC have helped us tremendously.”

Imagine how you would feel if you found out that your child was born with a degenerative, incurable disorder that would affect their quality of life. Now, imagine that they never received that diagnosis, delaying their access to life-changing treatment by years.

When you donate to MDC, you can help influence leaders to address gaps in our healthcare system, giving people living affected by a neuromuscular disorder a voice and improving their quality of life.

“The work that MDC put into advocating for SMA newborn screening is so important to so many families, and the only reason my family’s life is the way it is today,” Taylor says. “Many families do not have knowledge of the diagnosis or don’t know what they will need in order to give their children the best options in life. I cannot express the amount of appreciation I have for the work MDC put into getting SMA included in newborn testing!”

MDC is working hard to bring positive change to our community and to support the next generation of Canadians. But we need your help.

It takes just one generous act to change our country – and our future – for good.

Donate now!