Donors make it possible to invest in both neuromuscular disorder research and care, now and for the future

FOR IMMEDIATE RELEASE – Muscular Dystrophy Canada (MDC) together with the Neuromuscular Disease Network for Canada (NMD4C) are thrilled to announce the recipients of the National Clinical and Post-doctoral Fellowships funding competition. This competition involves an extensive review by leading Canadian neuromuscular researchers and clinicians to ensure the top-ranked candidates are selected for this funding opportunity.

“Too often we hear from our community about the long and difficult process of accessing specialists or that they are unable to find information on their neuromuscular disorder, let alone access to treatments or trials. We know how critical it is to invest in the future generations of neuromuscular disorder specialists to drive research momentum forward and improve the current standard of care available in Canada,” said Stacey Lintern, CEO, Muscular Dystrophy Canada. “Together, by providing both Post-doctoral and Clinical Fellowships, we are ensuring the sustainability and progress of Canadian neuromuscular research as well as building a stronger infrastructure of skilled clinicians educated on neuromuscular disorders in Canada.”

These fellowships are made possible each year by the outstanding community of supporters who work tirelessly to support the neuromuscular community. Generous donors, passionate fundraisers and dedicated Fire Fighters across the country make it possible for MDC to fund promising researchers, ensuring Canada is prepared to provide access to new life-changing treatments, while also filling the immediate need for advanced treatment and care options by supporting future clinicians. This is a community effort and we are extremely grateful for the ongoing support. From the entire neuromuscular community, congratulations to all recipients!

Post-doctoral fellowship recipients:

Dr Yassine Ouhaddi
Centre de recherche du CHU Sainte-Justine
Montreal, QC

Dr Cedric Happi-Mbakam
SickKids Hospital
Toronto, ON

Clinical fellowship recipients:

Dr Yiu-Chia Chang
University of Western Ontario
London, ON

Dr Mark Krongold
University of British Columbia
Vancouver, BC

Dr Bram De Wel
University of Calgary
Calgary, AB

MDC and NMD4C extend special thanks to the Canadian Society of Clinical Neurophysiologists (CSCN) for jointly funding a clinical fellow this year.

Learn more about the fellowship awards

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ABOUT MUSCULAR DYSTROPHY CANADA

Muscular Dystrophy Canada’s mission is to enhance the lives of those impacted 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 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

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

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

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

Neuromuscular Disease Network for Canada Awarded 5-Year Grant from CIHR-IMHA and Funding from MDC to Strengthen Canadian Neuromuscular Research and Care

Neuromuscular Disease Network for Canada Awarded 5-Year Grant from CIHR-IMHA and Funding from MDC to Strengthen Canadian Neuromuscular Research and Care

For Immediate Release: June 21, 2023

Toronto, ON – The Neuromuscular Disease Network for Canada (NMD4C) has received a network grant from the Canadian Institutes of Health Research – Institute of Musculoskeletal Health and Arthritis (CIHR-IMHA), providing funding of $200 000 per year for five years – with matched funding from Muscular Dystrophy Canada (MDC) – to strengthen the care, research and treatment of neuromuscular diseases (NMDs) for all Canadians. The new grant brings together an expanded group of clinicians, scientists and patient representatives under the leadership of Dr Hanns Lochmüller (Children’s Hospital of Eastern Ontario) and Dr Homira Osman (MDC).

“Since 2020, NMD4C has made remarkable progress in uniting Canada’s neuromuscular community. In these rare diseases, it’s crucial that we work together. We’re delighted that we have grown to more than 500 members spanning multiple disciplines, sectors, and areas of expertise. The network has made tremendous strides in building capacity through training and education, providing leadership and advocacy to improve access to approved novel treatments, and strengthening research resources and infrastructure,” explains Dr. Hanns Lochmüller, Senior Scientist, CHEO Research Institute and Professor of Neurology, University of Ottawa. “But there’s still so much more to be done. New scientific challenges and opportunities mean that networking across Canada and the globe is even more important. This new funding will allow us to expand our supportive, collaborative, networked community of neuromuscular stakeholders, bringing together an unparalleled concentration of NMD expertise to provide a Canada-wide platform for communication, collaboration, and best-practice sharing. We’re very grateful to CIHR-IMHA and MDC for the funding that allows us to continue our work. We have some really exciting plans for this next phase of the network, particularly for our young doctors and researchers, and we can’t wait to get started.”

Building on the NMD4C’s successful work over the past three years, this new grant will enable an ambitious new program of research, networking, and clinical transformation to address emerging challenges in the field of NMDs. With a total of 67 named co-investigators from across the country bringing in their wider teams of researchers, this is one of the largest networking projects in the rare disease field in Canada. Matching funds are being provided by Canada’s leading NMD advocacy organization and partner on the grant MDC, for a combined total amount of $400 000 per year of network funding.

“We greatly value our longstanding partnership with the CIHR, and are deeply thankful to our many partners, Fire Fighters, donors, volunteers and supporters who generously give so that we can fund the vital work of NMD4C,” said Dr Homira, Osman, Vice-President, Research and Public Policy, Muscular Dystrophy Canada. “In addition to offering matched funding, our role in the new grant will be to ensure evidence is translated into practices and policies that will make a tangible difference in the lives of Canadians affected by neuromuscular disorders. Aligned with our recently unveiled Breaking Down Barriers five-year strategic direction, we will partner closely with researchers, clinicians, and the neuromuscular community to enhance proactive and collaborative approaches focused on strengthening infrastructure, enhancing capacity, establishing centers of excellence, and facilitating the dissemination of research outcomes.”

This new grant unites people with lived experience, knowledge users, clinicians, and researchers to execute a new research plan with the following objectives:

Learn more “Neuromuscular Disease Network for Canada Awarded 5-Year Grant from CIHR-IMHA and Funding from MDC to Strengthen Canadian Neuromuscular Research and Care”

Abilities (at the) Centre of new location for Walk and Roll for Muscular Dystrophy Canada

FOR IMMEDIATE RELEASE June 16, 2023

Toronto, Ontario – The 2023 Walk and Roll for Muscular Dystrophy Canada (MDC) has changed venues from a local park to the Abilities Centre in Whitby in an effort to ensure the event is as accessible as possible for all participants. It takes place on June 25, 2023.

“MDC is committed to breaking down barriers for the neuromuscular community. When we determined that the park location originally selected to host the Durham Walk and Roll for Muscular Dystrophy Canada posed accessibility issues for individuals using mobility devices, we set out to find a new site,” said Stacey Lintern, CEO, Muscular Dystrophy Canada. “We couldn’t be happier to be partnering with the Abilities Centre. It is the perfect space for the Walk and Roll event, and our community.”

Walk and Roll for Muscular Dystrophy Canada is MDC’s signature fundraising event, raising funds to provide Canadians affected by neuromuscular disorders support programs and services, invest in ground-breaking research and advocate for better public policies. Thirty-three events are taking place across Canada with an overall goal of raising $1.3 million.

The Abilities Centre strives to make communities more accessible and inclusive to increase the quality of life for every individual and enable them to participate fully in community life. They offer a wide range of programs and services, and lead research and advocacy on inclusion issues.

“Events like the Durham Walk and Roll for Muscular Dystrophy Canada are so important to bring attention to, and raise funds for, the organizations that are working tirelessly to make the communities where we live, work, and play more accessible and inclusive,” said Ross Ste-Croix, General Manager & COO of Abilities Centre.

Previously known as the Walk for Muscular Dystrophy, the nationwide fundraising event also underwent a name change this year to be more inclusive and reflective of the neuromuscular community. Anyone interested in participating in the Durham Walk and Roll for Muscular Dystrophy Canada can register at walkrollmdc.ca

The Durham Walk and Roll for MDC fundraising goal for 2023 is $53,000. Support your community by making a donation here.

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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.

ABOUT ABILITIES CENTRE

Abilities Centre exists to unlock potential through accessibility. Whether through vocational services, therapeutic recreation, adult day programs, or sport and recreation, every program and service at Abilities Centre is aimed at re-imaging how Canadians live, work and play. We continue to focus our impact to build towards an inclusive, barrier-free life for all Canadians. For more information abilitiescentre.org

FOR MORE INFORMATION CONTACT:

Heather Rice
Heather.Rice@muscle.ca
902-440-3714

FUNDING FOR INNOVATIVE THERAPIES FOR HEREDITARY ATAXIAS

For immediate release

Montréal, April 28, 2023 – It is with great enthusiasm that Génome Québec, Ataxia Canada and Muscular Dystrophy Canada announce the launch of the Innovative Therapies for Hereditary Ataxias competition to encourage the discovery and development of new therapies and to mobilize the research ecosystem. The three organizations aim to jointly invest $1M to support projects with a maximum of $330,000 per project for a period of two years.

The goal of this competition is to meet the needs of people affected by these rare neurological diseases, to enable them to benefit from genomic innovations and to encourage the development of innovative therapies. It supports the objectives and orientations of the provincial and federal governments, notably by accelerating therapeutic innovation (Politique québécoise pour les maladies rares) and by supporting research to improve access to affordable and effective drugs for the treatment of rare diseases.

This competition is open to researchers affiliated with a Québec university and its affiliated institutions (including hospitals and research institutes).

The deadline for submitting the mandatory registration to Génome Québec is June 8, 2023.

Learn more “FUNDING FOR INNOVATIVE THERAPIES FOR HEREDITARY ATAXIAS”

Celebrating outstanding commitment through the Dr David Green Awards

These individuals show extraordinary commitment to raising funds, increasing awareness and advocating for the neuromuscular disorder (NMD) community, supporting continued research, engaging other community members and building positive connections.

Volunteers are the backbone of our organization. They share creative ideas, give their time, and contribute their experiences to help advance advocacy efforts, push research forward, and deliver essential programs and services. MDC is very grateful to have such amazing individuals and groups committed to supporting our mission and the Canadian NMD community.

Learn more about our 2023 award winners.

MDC funds nine new projects to propel research forward and break down barriers

Toronto, Ontario – Muscular Dystrophy Canada (MDC) is proud to announce an investment of $832,766 to fund nine new clinical and translational science research projects in 2023 through the MDC neuromuscular disorder (NMD) research grant competition, a dedicated Canadian source of funding for neuromuscular research.

“Muscular Dystrophy Canada is a leader in funding ground-breaking research for all neuromuscular disorders. This does two things: first, it lets us advance the global understanding of NMDs and the advancement of more treatments, therapies, and potential cures. And second, our involvement with these research projects means we can get people the most up-to-date information they need,” stated Stacey Lintern, CEO, Muscular Dystrophy Canada.

Drawing on the expertise of Canadian and international reviewers, including neurologists, researchers, allied health care professionals, and people with lived experience, the top projects were identified for funding.

“We held a rigorous peer-review process, and as a result, the projects selected for funding are diverse and potentially high-impact research. The projects cover different neuromuscular disorders and aspects of diagnosis, clinical care, and management and evaluate the impact on muscle function and systems outside of muscles, as well as quality of life,” said Dr. Homira Osman, Vice President, Research and Public Policy, Muscular Dystrophy Canada. She added, “supporting such projects not only contributes to the knowledge base, but it strengthens the Canadian neuromuscular research infrastructure and brings us one step closer to cures.”

The projects submitted this year for consideration were of remarkable quality, and we thank each and every team for their interest in doing this important work. MDC relies on the generosity of Fire Fighters, donors and volunteers to invest in life-changing research, and is honoured to fund the exceptional and bright researchers, clinicians and academics who will lead these nine new projects taking place in hospitals and universities across Canada.

Learn more “MDC funds nine new projects to propel research forward and break down barriers”