News – Page 5 – Muscular Dystrophy Canada

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”

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

First of its kind: New program will increase patient engagement in neuromuscular disorder research

Toronto, Ontario – Muscular Dystrophy Canada (MDC) and the Neuromuscular Disease Network for Canada (NMD4C) are proud to announce the launch of imPORTND, an online patient-oriented research (POR) training platform.

The imPORTND platform offers online, self-directed, free training modules on how to conduct research that is informed by, and most meaningful to individuals living with neuromuscular disorders. The modules are designed for all members of the study team: including researchers, lab staff, and patient- and family partners.

“Canadians affected by neuromuscular disorders and their families are increasingly asked to take an active role in research, but they often feel unprepared to participate,” said Dr. Homira Osman, Vice President, Research and Public Policy, Muscular Dystrophy Canada. “The training provided through imPORTND will ensure people have the confidence and knowledge needed to be involved in all stages of research: decision-making, design, planning, delivery, evaluation and knowledge mobilization. The modules will help ensure patient and family partners are well-equipped to share their lived expertise, guide discussions and inform decisions regarding health care, research, and therapy development with policymakers, industry and scientists.”

Focused on a ‘Nothing about me; without me’ patient-centred approach, imPORTND was co-developed with a team of expert patient partners, clinicians, researchers, and patient partner organization members, and is the first POR training developed specifically for neuromuscular disorder research.

“When patients and researchers collaborate on projects, the work they co-create is more meaningful and able to make real and positive impacts. Learning to work together is what ‘imPORTND’ is all about. The modules focus on the tools, skills and relationships needed by researchers, patient-partners and clinicians to partner for work that is more inclusive, equitable, diverse and accessible.,” said Dr. Kathryn Selby, Medical Director, Pediatric Neuromuscular Program of BC and the Yukon and Patient-Oriented Resources Lead Investigator at the Neuromuscular Disease Network for Canada.

The neuromuscular community is excited to participate in projects that impact them directly.

“As patient partners, our voices give deeper understanding to neuromuscular conditions, with each one of us sharing our unique lived experiences. Together with medical research teams, we prioritize meaningful timely research that immediately serves to improve quality of life,” said Linda Niksic, NMD4C Patient Research Partner, and contributor to the imPORTND training modules.

Additional information about imPORTND, can be found at importnd.neuromuscularnetwork.ca.

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ABOUT MUSCULAR DYSTROPHY CANADA

MEDIA CONTACT

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

$360,000 in fellowships awarded to advance neuromuscular research and healthcare

FOR IMMEDIATE RELEASE January 19, 2023

Muscular Dystrophy Canada (MDC) together with the Neuromuscular Disease Network for Canada (NMD4C) are excited to announce the recipients of a funding competition, which awarded national post-doctoral fellowships in neuromuscular research and clinical fellowships in neuromuscular medicine and electromyography.
This competition saw many incredible applicants and involved a thorough and extensive review by leading Canadian neuromuscular researchers and clinicians to ensure the most promising and top-ranked researchers, and leaders in neuromuscular care, received funding.

“This initiative helps train and educate the next generation of neuromuscular researchers and clinicians. The post-doctoral fellowships help strengthen neuromuscular research capacity, and the clinical fellowships will strengthen the specialist care available to the neuromuscular community,” said Stacey Lintern, CEO, Muscular Dystrophy Canada. “For our clients, that means we are investing in the sustainability of neuromuscular research, which brings us one step closer to finding cures, and in skilled clinicians that will provide clinical care to individuals affected by neuromuscular disorders and help raise standards of care.”

Learn more “$360,000 in fellowships awarded to advance neuromuscular research and healthcare”

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.”