Advocacy – Page 4 – Muscular Dystrophy Canada

Funding for first-ever National Strategy for Drugs for Rare Diseases announced

FOR IMMEDIATE RELEASE March 22, 2023

Toronto, Ontario – Muscular Dystrophy Canada applauds the Government of Canada for committing $1.5 billion over three years in support of a National Strategy for Drugs for Rare Diseases to help increase access to, and affordability of, promising and effective drugs for rare diseases to improve the health of patients across Canada.

“MDC works with Canadian Fire Fighters, donors, volunteers and other like-minded organizations to break down barriers for the neuromuscular community. The major barriers that exist for our diverse community, which consists of individuals with rare and ‘ultra-rare’ neuromuscular disorders, are lack of cures and delayed – out of reach treatments. So this announcement is great news for our community,” said Stacey Lintern, CEO, Muscular Dystrophy Canada. She added, “we are excited that this announcement has been made but time is of the essence for many rare diseases and the sooner we can move this from strategy to action, the better it is for our community.”

This long-awaited announcement identified four areas of focus for the national strategy and funding support for provinces and territories to improve access to new and emerging drugs, enhance access to existing drugs, early diagnosis, and screening for rare diseases.

“The areas of focus align perfectly with the work that MDC is already doing such as ensuring early diagnosis and clinical genetic testing. We know access to an accurate diagnosis, coupled with early access to treatments, are important for optimal outcomes and better quality of life,” said Lintern.

“MDC welcomes a strategy where the patient and family are at the forefront and that supports an evidence-based care journey. We are committed to working with our partners like the Neuromuscular Disease Network for Canada (NMD4C) to help make that happen, and bring about better diagnostic tools, support for clinical trials and evidence that supports decision-making. We look forward to a time when the neuromuscular community has access to accurate and early diagnoses, life-changing treatments, and supports regardless of where they live, their age or the rarity of the condition.”

The full Government of Canada announcement can be read here

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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

A Partnership to Fund Research for Hereditary Ataxias

FOR IMMEDIATE RELEASE

Montreal, February 28, 2023 – It is with great pleasure that Génome Québec, Ataxia Canada and Muscular Dystrophy Canada join forces in order to create a funding program for research on hereditary ataxias.

The goal of this program is to meet the needs of people affected by these rare neurological diseases, to allow them to benefit from genomic innovations and to encourage the development of innovative therapies. This program would support the objectives and orientations of the federal and provincial governments, notably by accelerating therapeutic innovation (Politique québécoise pour les maladies rares) and by presenting a concrete alternative for the creation of new drugs for the treatment of rare diseases (Building a National Strategy for Drugs for Rare Diseases: What We Heard from Canadians).

This partnership will not only mobilize the research community in the field, but also undertake a co-development approach in collaboration with foundations representing people affected by ataxia. This alliance will thus contribute to the democratization of research while increasing public awareness of ataxias.

This program will only be open to Québec researchers; more information will be available in the spring.

Learn more “A Partnership to Fund Research for Hereditary Ataxias”

MDC, CNDR, NMD4C partner to ensure Canadians can access FSHD cure(s)

FOR IMMEDIATE RELEASE February 28, 2023

Toronto, Ontario – Canadians affected by facioscapulohumeral muscular dystrophy (FSHD) could face substantial delays in accessing clinical trials and any Health Canada approved life-changing treatments and therapies when they become available. Muscular Dystrophy Canada (MDC), the Canadian Neuromuscular Disease Registry (CNDR), and the Neuromuscular Disease Network for Canada (NMD4C) are joining forces to help prevent that from happening.

“Studies show that Canadians have less frequent and timely access to therapies for rare diseases, like FSHD; that very few therapies approved elsewhere in the world are even submitted to Health Canada for regulatory approval; and, that if treatment is approved there is a vast difference in who has access, across the provinces,” said Stacey Lintern, CEO Muscular Dystrophy Canada.

Learn more “MDC, CNDR, NMD4C partner to ensure Canadians can access FSHD cure(s)”

Community Statement: Update on Deflazacort®/Calcort® Availability

February 24, 2023 – In late January, we became aware of a possible Deflazacort®/Calcort® supply and access issue in Canada. Deflazacort is the most commonly used corticosteroid prescribed in Canada and an affordable option for our Canadian Duchenne muscular dystrophy community. We learned that the problem was primarily due to manufacturer and distributor changes and a need to increase the price.

Muscular Dystrophy Canada and Defeat Duchenne Canada partnered to resolve the issue quickly so a shortage could be avoided, and individuals wouldn’t have to potentially switch to prednisone (as this was a concern brought to us by families). At the same time, together, we worked directly with families to better understand the issue at a local (pharmacy) level.

While the impact did not become widespread, we know of a few Duchenne patients and families directly impacted.

We are pleased to report that a shipment of Deflazacort®/Calcort® arrived last week, and inventory is available for the entire country. There is a price increase (approximately $50 for 60 pills) and a change in format (pills in a box vs. sleeve format).

The new manufacturer has confirmed that the backlog in the market has been solved and will provide this critical product to the Canadian market from now on. We will continue to follow this issue closely as we recognize how vital Deflazacort is for people with Duchenne muscular dystrophy. We look forward to ensuring a sustainable supply of Deflazacort and good access to other approved drugs prescribed for managing and treating neuromuscular disorders in Canada.

If you are still experiencing challenges accessing Deflazacort®/Calcort®, please contact us at research@muscle.ca or call 1-800-567-2873 ext. 1114.

Thank you,

Stacey Lintern
Chief Executive Officer
Muscular Dystrophy Canada

Perry Esler
Executive Director
Defeat Duchenne Canada

Neuromuscular disorders do not discriminate. Nor do they take a break over the holiday season.

Canadians affected by neuromuscular disorders celebrate a variety of different holidays over the winter season, however, amongst this diversity, our community has one thing in common: Often this time of year has added pressures for those living with neuromuscular disorders and their families.

Regardless of where you live in Canada, accessibility issues are amplified in the winter months. Whether it’s the winter storm and unplowed sidewalks that keep you home or the lack of accessible transit making it impossible to celebrate the way you had hoped, those living with neuromuscular disorders can lean on their Muscular Dystrophy Canada (MDC) Service Specialist for support. Service Specialists help navigate individual questions, complex decisions, advocate for change and provide emotional support during difficult moments.

And, thanks to you «our donors» we can answer each call and make a difference together.

Today, I hope you will make a gift to ease some of the pressure of the season for the thousands of individuals and their families, who turn to MDC as a trusted resource and partner in their neuromuscular journey.

Donate Today!

Learn more “Neuromuscular disorders do not discriminate. Nor do they take a break over the holiday season.”

Make an impact for someone like Brody today

As a baby, Brody wobbled when he sat unassisted, and struggled to control his legs when pulling himself into a standing position. Kelli, his mom knew something was not quite right.

And, at 20 months old, Brody saw his first specialist – the start of a three-year journey in search of his diagnosis. After meeting with half a dozen doctors, completing countless medical tests, and travelling hundreds of kilometres across Alberta, Brody was finally diagnosed with Duchenne muscular dystrophy at five years old.

Kelli says, “I don’t think any parent can prepare themselves to hear that their child has a progressive, incurable disease with no remission. It’s the most helpless feeling in the world!”

Through MDC Kelli, Brody and their family now have access to the resources they need. Brody’s school has hosted learning and educational sessions for teachers and classmates with MDC as their partner and as a family, they join our retreats to meet other families affected by Duchenne muscular dystrophy.

Donors make MDC’s support of families possible. Your gift today will provide much-needed support and resources to your neighbours in their time of need.

MAKE YOUR GIFT TODAY

Your generosity today means that MDC clients can count on us for supportive programs and services, while we continue to fund research and champion advocacy efforts on their behalf.

Because of you, we can partner with families in your community who need it most.

The gift you make today will change lives.

DONATE NOW

Give someone like Elvin the gift of independence

At four years old, Elvin had great difficulty walking and fell down frequently. Concerned, his parents took him to a specialist in Toronto, where he was diagnosed with Duchenne muscular dystrophy.

The youngest of four children, Elvin relies heavily on his family for support with daily activities including bathing, and going up and down stairs. His mom, Doreen has been one of his biggest supporters and champions.

Then, a few years ago, Doreen had a stroke. Devastating to the family, the effects of the stroke mean that Doreen is unable to support Elvin as his primary caregiver. Looking for help, they registered with Muscular Dystrophy Canada (MDC).

Through MDC’s life-changing programs, Elvin now has access to a wheelchair lift allowing him to come and go without relying on his family to carry him up and down stairs. This lift lets Elvin be more independent and gives his family a break from the physical demands of caring for him.

Your gift today will enable MDC to continue helping families, like Elvin’s, enjoy greater independence, and improved quality of life.

MAKE YOUR GIFT TODAY

Your generosity today means that MDC clients can count on us for supportive programs and services, while we continue to fund research and champion advocacy efforts on their behalf.

Because of you, we can partner with families in your community who need it most.

The gift you make today will change lives.

DONATE NOW

Support someone like Elude today

For years, Elude complained of pain in his legs that would not go away and after several doctor’s visits, he was diagnosed with oculopharyngeal muscular dystrophy. As his disease progressed, it became increasingly difficult for Elude to complete everyday tasks and his wife, Gaétane stepped in to support.

For more than 30 years, Gaétane has struggled to take care of everything on her own including completing daily chores, caring for Elude and maintaining their home in New Brunswick. Then eight years ago, Elude’s condition deteriorated. Thankfully, one of their nurses told them about Muscular Dystrophy Canada (MDC).

“We asked for help to purchase equipment including a machine that helps to clear Elude’s throat, and mobility devices like a walker and a chair lift,” Gaétane said. Having access to these devices greatly improves Elude’s mobility, giving him greater enjoyment and quality of life.

MDC also supports caregivers, like Gaétane, who receives a few hours of domestic help each week, giving her a much needed break to recharge. Those few hours ensure she is her best self when at home with her husband.

Your gift today will enable MDC to continue delivering life-changing programs that help families, like Elude and Gaétane, enjoy greater independence and an improved quality of life from the comfort of their own home.

MAKE YOUR GIFT TODAY

Your generosity today means that MDC clients can count on us for supportive programs and services, while we continue to fund research and champion advocacy efforts on their behalf.

Because of you, we can partner with families in your community who need it most.

The gift you make today will change lives.

DONATE NOW

Make a life-changing difference for someone like Lucas

At eight years old, Lucas was diagnosed with centronuclear myopathy (MTM1), which causes severe muscle and lung weakness and greatly affects his mobility. Because of his disorder, Lucas uses a wheelchair, and relies heavily on his family, especially his mom and dad, Sarah and Arnaud, for support with daily activities including personal care and going up and down stairs.

As Lucas got older, and his disorder progressed, it became more difficult for Sarah to lift him to the bathroom or carry him upstairs to his bedroom. Left with the difficult task of modifying their home in Quebec to meet Lucas’ needs, they registered with Muscular Dystrophy Canada (MDC), who stepped in to support these much-needed renovations.

With support from MDC, Sarah and Arnaud equipped their home with an elevating platform and a power lift, providing Lucas with improved mobility and quality of life.

Through MDC’s life-changing programs, Lucas can now move around his home freely without needing to rely on his family as much. This equipment lets Lucas be more independent and gives his family a break from the physical demands of caring for him.

Your gift today will enable MDC to continue delivering life-changing programs that help families like Lucas, Sarah and Arnaud, enjoy greater independence, and improved quality of life.

MAKE YOUR GIFT TODAY

Your generosity today means that MDC clients can count on us for supportive programs and services, while we continue to fund research and champion advocacy efforts on their behalf.

Because of you, we can partner with families in your community who need it most.

The gift you make today will change lives.

DONATE NOW

Canadians like Johnny need your support now

When Johnny began experiencing weakness and muscle deterioration, he brushed it off as a sign of aging. As an athlete, who enjoyed running and golfed regularly, being diagnosed with Limb-Girdle muscular dystrophy in his late 40’s was life changing for him and his wife, Carla.

At first, Johnny’s biggest issue was climbing stairs, until his disorder had progressed to the point where he not only couldn’t climb stairs anymore but, also had great difficulty standing up and sitting down. Left with the difficult task of modifying their home in British Columbia to meet Johnny’s needs, they registered with Muscular Dystrophy Canada (MDC).

With support from MDC, Carla and Johnny were able to equip their home with several ramps. MDC also provided Johnny with leg braces, which Carla says, “he wears all the time! The leg braces provide him with a lot of support and enable him to do things that he otherwise wouldn’t be able to do.”

Carla and Johnny feel grateful for the support of MDC. Carla says, “all interactions that Johnny has with Sharon [MDC’s Service Specialist in British Columbia] are so kind and supportive. She listens to him, and is so caring. It’s like they’re friends. There is no cure for muscular dystrophy and although we have wonderful doctors, there is no other support for us, other than MDC!”

Your gift today will enable MDC to continue delivering life-changing programs that help families like Johnny and Carla, enjoy greater independence, and improved quality of life.

MAKE YOUR GIFT TODAY

Your generosity today means that MDC clients can count on us for supportive programs and services, while we continue to fund research and champion advocacy efforts on their behalf.

Because of you, we can partner with families in your community who need it most.

The gift you make today will change lives.

DONATE NOW