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”

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”

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

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.

MEDIA CONTACT

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

International research competition results in funding for innovative Collagen 6- related dystrophy research projects

Muscular Dystrophy Canada (MDC) is pleased to announce two Collagen 6- related dystrophy (Col6-RD) research projects will be funded thanks to generous donors, and a partnership with Cure CMD (Congenital Muscular Dystrophy).

Collagen 6- related dystrophies (Col6-RD) are genetic types of muscular dystrophies that include Bethlem myopathy, Ullrich congenital muscular dystrophy and intermediate forms (Limb-girdle muscular dystrophy R22/D2). They are characterized by muscle weakness that can begin in infancy and can affect mobility, breathing, eating and activities of daily living.

Currently there is no cure for Col6-RD and knowledge on how the disorder develops is limited. Research is the essential next step towards therapy development.

Learn more “International research competition results in funding for innovative Collagen 6- related dystrophy research projects”

Announcing Muscular Dystrophy Canada’s 2022 Research Grant Competition winners!

We are pleased to share that we are funding nine new clinical and translational science research projects focusing on spinal muscular atrophy, Duchenne and Becker muscular dystrophies, immune mediated neuropathies, congenital myasthenic syndromes, immune-mediated neuromuscular junction disorders, myotonic dystrophy, occulopharyngeal muscular dystrophy, spinal bulbar muscular atrophy (Kennedy disease), and other related neuromuscular disorders.

Thank you to our donors who helped make this possible and to our Research Grant Review Panels of researchers, clinicians and community members with lived experience who volunteered their time to carefully review and help narrow down applications to our final projects.

“Through these exciting projects, these talented and dedicated Canadian researchers will develop new methods for disease diagnosis, uncover novel insight into disease pathogenesis, and develop new treatments options, amongst other meritorious projects,” says Dr. Robin Parks, Chair of the Medical and Scientific Advisory Committee for Muscular Dystrophy Canada (MDC) and Chair of the Translational Science Review Panel. “Through their support of MDC, donors, patients and their families are helping fund outstanding research projects that will move neuromuscular research forward and ultimately improve care and quality of life for those affected by neuromuscular disorders – which aligns with MDC’s mission.”

“We are extremely pleased with breadth of proposals received to our call for clinical and translational science research proposals we received this year. This is a hopeful sign of the continued momentum and progress in neuromuscular research. The nine selected recipients show exemplary innovation, dedication and promise of potential impact for a wide range of neuromuscular disorders,” says Stacey Lintern, CEO of Muscular Dystrophy Canada. “Because of our incredible community, we are able to fill a critical gap in research funding. We know how much change is possible from Canadian researchers and we are continually working with our supporters to ensure we invest in meaningful ways.”

Thank you to the Canadian neuromuscular research community for putting forward impactful applications.

2022-2023 WINNERS:

  • Dr. Thomas Durcan, MD, The Royal Institution for the Advancement of Learning/McGill University
    Building a screenable human 3D neuromuscular junction model for neuromuscular disorders
  • Dr. Christopher Perry, MD, York University
    Mitochondrial-targeted therapies to improve Duchenne muscular dystrophy outcomes
  • Dr. Craig Campbell, MD, London Health Sciences Centre Research Inc. (Lawson Health Research Institute)
    Genome-wide DNA methylation profile in Duchenne Muscular Dystrophy
  • Dr. Michael Berger, MD, University of British Columbia
    Measuring balance in chronic inflammatory demyelinating polyneuropathy
  • Dr. Bernard Jasmin, MD, University of Ottawa
    Endurance training as a novel therapeutic strategy for Myotonic Dystrophy type 1
  • Dr. Jodi Warman-Chardon, MD, Ottawa Hospital Research Institute
    QP-OPMD: Quantitative MRI Imaging to Assess Progression in Oculopharyngeal Muscular Dystrophy
  • Dr. Kerri Schellenberg, MD, The University of Saskatchewan
    Exploring the impact of Spinal Bulbar Muscular Atrophy on persons self-identifying as Indigenous
  • Dr. Reshma Amin, MD, The Hospital for Sick Children Dr. Aaron Izenberg, MD, Sunnybrook Health Sciences Centre
    The Transition of TEENagers with Spinal Muscular Atrophy to a Multi-Disciplinary Adult Program
  • Dr. Maryam Oskoui, MD, The Research Institute of the McGill University Health Centre
    Motor network connectivity in spinal muscular atrophy: new pathways for intervention

WATCH THE ANNOUNCEMENT

Post-doctoral Fellowships to advance neuromuscular research

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. This competition saw many incredible applicants and involved a thorough and extensive review by leading Canadian neuromuscular researchers to ensure the most promising and top-ranked researchers received the funding.

The fellowship provides salary support for early-career scientists to conduct postdoctoral studies in a laboratory focused on research of NMDs. Successful applicants will receive $40,000 in funding over a one-year period.

“This initiative will assist with training and education for the next generation of neuromuscular researchers and strengthens the biomedical infrastructure to build neuromuscular research capacity in Canada. For our clients, this means we are investing in the sustainability of neuromuscular research which brings us one step closer to finding cures.”

Stacey Lintern, CEO of Muscular Dystrophy Canada

Post-doctoral research fellowship recipients:

Emanuela Pannia, PhD

Research Institute, Hospital for Sick Children

Post-doctoral Supervisor: James Dowling, MD, PhD, Clinician and Senior Scientist, The Hospital for Sick Children

Title of Research: Determining the molecular mechanisms and environmental modifiers of progressive liver disease in X-linked myotubular myopathy.

Overview: The goal of this study is to identify the cause of liver disease in X-linked myotubular myopathy (XLMTM) and the effects of diet and the immune system on its development. This study will be the first to identify causes of liver disease in XLMTM and discover new therapies to help children with this devastating disease.

Talita Conte, PhD

Centre Hospitalier Universitaire Sainte-Justine

Post-doctoral Supervisor: Nicolas Dumont Pht, PhD , Assistant Professor, Centre Hospitalier Universitaire Sainte-Justine

Title of research: Novel strategy targeting dysfunctional muscle stem cells to mitigate Myotonic dystrophy type 1

Overview: The goal of this study is to provide important advancements on the new field of eliminating defective stem cells in Myotonic dystrophy type 1 (DM1) cell cultures, which would restore the function of the remaining muscle stem cells. This advancement would open a new option to DM1 patients.

 

These fellowships exemplify the excellence of future generations of researchers. Generous supporters of Muscular Dystrophy Canada made this funding competition possible. Thank you for making a difference and investing in the future of neuromuscular research.

NMD4C propelling research forward for patients, scientists and clinicians

FOR IMMEDIATE RELEASE – Friday, January 31, 2020

Toronto, Ontario – A new partnership between Muscular Dystrophy Canada (MDC) and the Canadian Institutes of Health Research (CIHR) announces the creation of a new neuromuscular network in Canada.

Led by Dr. Hanns Lochmüller, NMD4C is an integrated research network for patients, scientists, and clinicians to improve outcomes and access to therapies for patients with neuromuscular disorders in Canada.

“We’re absolutely delighted to receive this catalyst funding for our NMD4C network. Because neuromuscular diseases are individually rare, but collectively affect many children and adults across Canada. Working together, pooling expertise and developing shared infrastructure at a national level is essential if we want to make rapid progress towards giving all Canadian patients world-class care and develop new treatments. This is a fantastic opportunity for doctors, researchers and the patient community to come together to do just that, and we can’t wait to get started,” stated Dr. Lochmüller.

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Key topics in Spinal Muscular Atrophy research discussed at first ever Muscular Dystrophy Canada SMA Research Summit

Toronto, November 15, 2019 – Research experts, the medical community, and industry stakeholders came together in Toronto this week at the inaugural Muscular Dystrophy Canada (MDC) SMA Research Summit to discuss new research and development.

“This summit was an important opportunity for us to review the latest developments and discuss where there are opportunities to enhance our role in the neuromuscular community in order to provide the proper support for individuals and families impacted by SMA,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We thank our generous sponsors Biogen, Novartis, and Roche for providing us with the opportunity to have these important conversations, as well as our organizing committee—co-chairs Dr. Rashmi Kothary and Dr. Maryam Oskoui, as well as Dr. Craig Campbell and Dr. Lawrence Korngut.”

Key topics under discussion included new research, clinical trial developments, and the changing treatment and regulatory landscape in Canada.

“As part of MDC’s ongoing commitment to influence positive change, we convened leading medical and scientific experts to share and collaborate in ways that will foster medical advances and impact the lives of the individuals and families that we serve. We are excited to continue the momentum and are planning MDC’s first nationwide Neuromuscular Impact Conference, which will be held next year so our clients have an opportunity to hear and speak to the scientific and medical community,” said Daria Wojtal, Director of Research, Muscular Dystrophy Canada.

Spinal muscular atrophy is a severe, inherited, progressive neuromuscular disease that causes major problems with walking, muscle strength, fine motor skills, and the basic physical functions of breathing, swallowing, and feeding. Until recently, there were limited treatment options for SMA, but prognosis has been transformed with the recent availability of a number of effective disease-modifying therapies, notably nusinersen, known as Spinraza. Great advances have been made and there is a pipeline of clinical trials that are transforming what it means to be diagnosed with this rare neuromuscular disease. 

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.

MEDIA CONTACT

Heather Rice
Muscular Dystrophy Canada
Heather.Rice@muscle.ca

Alberta Fourth Province to Expand Access to Spinraza™ for Patients Impacted with Spinal Muscular Atrophy

Alberta fourth province to expand access to SPINRAZA™ for patients impacted with Spinal Muscular Atrophy.

Muscular Dystrophy Canada (MDC) commends the Government of Alberta for joining Quebec, Saskatchewan, and Ontario in expanding access to SPINRAZA™, a life-changing treatment for individuals impacted with Spinal Muscular Atrophy (SMA).

In Alberta, the following patients will now be eligible for reimbursement of SPINRAZA™, in addition to Type 1 patients:

  • patients who are pre-symptomatic with two or three copies of SMN2, or
  • have had disease duration of less than six months, two copies of SMN2, and symptom onset the first week after birth and on or before seven months of age, or
  • are under the age of 18 with symptom onset after six months of age, regardless of the ability to walk.
  • Other patients who do not meet the expanded funding criteria may be considered in exceptional cases.
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More Patients Impacted with Spinal Muscular Atrophy in Ontario to Gain Access to Spinraza™

Toronto, Ontario – Muscular Dystrophy Canada (MDC) applauds the Government of Ontario for expanding access to SPINRAZA™, a life-changing treatment for individuals impacted with Spinal Muscular Atrophy (SMA). In Ontario, expanded coverage of SPINRAZA™ will include the following, in addition to existing coverage for Type 1 patients:
  • patients who are pre-symptomatic with two or three copies of the SMN2 gene;
  • patients with a disease duration of less than six months, two copies of the SMN2 gene, and symptom onset the first week after birth and on or before seven months of age;
  • patients under the age of 18, with symptom onset after six months of age and who have never achieved the ability to walk independently.
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