News – Page 9 – Muscular Dystrophy Canada

Change someone’s tomorrow – today!

I had a fifty-fifty chance of inheriting the gene from my mother. I didn’t. But my little brother did. I feel a lot of guilt but all I can do is give my time, money or muscles – sometimes all three – to make a difference for others who are affected by neuromuscular disorders

William Truman

William Truman is a Fire Fighter from Quebec. He’s also a father, friend, son, brother and generous supporter of Muscular Dystrophy Canada (MDC).

Will and his brother Joe, separated by just a year and a half, grew up extremely close. Despite being diagnosed with Duchenne muscular dystrophy (DMD), Joe was always laughing and was such a happy little boy. As a protective older brother, watching Joe grow up with so many challenges was very difficult for Will and brought up a lot of feelings of guilt.

It’s hard not to get emotional when I hear stories like this from families affected by neuromuscular disorders. When Will and I spoke about why he supports MDC, I knew I had to share it with you.

But before I go any further, let me say thank you. It is because of the continued support of donors like you that we are seeing great change and momentum in neuromuscular research.

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Will longed to see research advances in Joe’s lifetime.

We are making progress, but there is still a long way to go before we find cures. I’m asking you to help us continue this momentum by making a donation today.

When Will was a kid, MDC helped his family purchase equipment and provided them with supportive services. Having MDC as a partner in Joe’s journey, their journey as a family, was so impactful.

That’s why Will feels that it’s so important to support MDC. He knows exactly how much you are helping families. Right now, and in the future. In his words:

It’s impossible to not find reward in supporting MDC. My favourite part is meeting the children who participate in events like the High Rise Challenge. Seeing their shy smiles, seeing it change to not so shy when they get to sit in the fire truck. That’s why I raise funds – to help moms and dads who are going through what my parents experienced so that their stories have a different ending.

The future is promising. In 2021, the first-ever gene therapy clinical trial for DMD is taking place in Canada and new therapies are in development. We know the breakthroughs we make in research are not isolated to one neuromuscular disorder and will lead to even more new trials and treatments.

Your unwavering support is critical as we work with research, clinical and academic communities to relentlessly search for treatments and cures.

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Your support will make the world a little brighter for families on this journey.

I am so grateful when supporters like Will share their story with the hopes of motivating others to give back as well. At MDC we are a powerful community and the impact we make is because of you.

I am so proud of how far we’ve come and where we’re going. Thank you for changing someone’s tomorrow – today!

Stacey Lintern
Chief Executive Officer

PS. Your gift today will help ensure families in the future will benefit from the emerging research as we get closer to cures.

PPS. Making your donation is easy. Simply click here or you can also call 1-800-567-2873 toll-free to make a gift by phone.

Recognizing dedication and passion beyond measure

Muscular Dystrophy Canada (MDC) recognizes and celebrates the contributions of individuals and groups who champion our mission with courage, determination, and passion through the Dr. David Green Awards Program. We are excited to announce the list of our esteemed 2021 award winners. These individuals show extraordinary commitment to raising funds, increasing awareness and advocating for the NMD community, supporting continued research, engaging other community members and building positive connections.

The type of compassion and leadership recognized by these awards is personified in Dr. Colleen O’Connell, MDC’s 2021 recipient of the Outstanding Healthcare Partner award. This award is presented to a physician or clinician for outstanding achievements in improving neuromuscular disorder clinical practice and/or who has played a major or lasting role in improving the quality of life of people affected by neuromuscular disorders and their families.

In the past year, Dr. O’Connell has supported MDC’s knowledge sharing and advocacy initiatives. Particularly, Dr. O’Connell supported MDC with gathering and sharing credible and timely information regarding COVID-19 and NMDs. She has advocated on behalf of patients with NMDs to receive prioritization for vaccination. She also presented at our #LetsTalkNMD webinars, and most recently, she is the chair of transition of care in DMD for an upcoming Duchenne conference. She has supported MDC in many ways and is exceptionally knowledgeable, available and willing to support the NMD community in Canada.

“MDC is incredibly grateful to have so many groups and individuals dedicated to supporting our mission and the NMD community as a whole,” says Stacey Lintern, CEO of Muscular Dystrophy Canada. “From giving their time, contributing innovative ideas, sharing their experiences and helping to advance MDC forward in all areas of our Mission efforts, volunteers are the back-bone of our organization. Thank you doesn’t even begin to cover just how appreciative we are of each and every one of you”.

We are so lucky to have such an amazing group of supporters, including Dr. O’Connell, who continue to support our Mission, challenge us to be better and unite the neuromuscular disorder community. Click here to learn more and see a full list of this year’s award winners.

2021 Award Winners

Providing a path to practice: fellowship funding to support top-ranked Canadian clinicians

Muscular Dystrophy Canada (MDC) together with the Neuromuscular Disease Network for Canada (NMD4C) and the Canadian Society of Clinical Neurophysiologists (CSCN) are excited to announce a funding competition to award three new national clinical fellowship awards in neuromuscular medicine and electromyography.

The awards provide salary support for early-career clinicians to participate in a neuromuscular medicine and electromyography training program, with successful applicants being eligible to receive up to $76,500 in funding over a one-year period.

“Through providing fellowship opportunities for residents to pursue high-quality clinical training in the field of neuromuscular medicine and electromyography, we will create a path to practice for talented clinicians with a passion for neuromuscular disease, raising the standard of treatment and access to care for all Canadians affected by neuromuscular disease,” said Dr. Hanns Lochmüller, Lead Investigator, NMD4C.

A total of three clinical fellowships will be awarded on a competitive basis for the 2022/2023 academic year. These fellowships have been made possible by generous supporters of Muscular Dystrophy Canada and an unrestricted educational grant from Grifols Canada.

“Finding a neuromuscular specialist is an important step in a person’s healthcare journey, and we know there are few specialists across the country. Physicians can foster an environment that supports effective and informed decision-making, which is central to person-and family centred care,” said Stacey Lintern, CEO, MDC. “Together with NMD4C and CSCN, we are building a pipeline of excellence, and investing in top-ranked clinicians that will provide clinical care to individuals affected by neuromuscular disorders and help raise standards of care.”

Competition details:

The competition opens on October 8, 2021 with a deadline for submissions of November 19, 2021. The fellowship is open to Canadian citizens or permanent residents who have completed postgraduate training in adult or paediatric neurology or physiatry in a Royal College of Physicians and Surgeons of Canada accredited program at the time of commencement of the fellowship (at least one Canadian citizen/permanent resident will be awarded funding). It is also open to International/Canadian applicants who have completed postgraduate training in adult or paediatric neurology or physiatry outside of Canada that have met the Royal College of Physicians and Surgeons of Canada requirements, if they have the support of the proposed Canadian fellowship training director. We encourage all who are eligible to apply!

View the full fellowship guidelines here.

Please share this opportunity with any of your colleagues and within your neuromuscular networks.

For further inquiries, please contact Dr. Homira Osman at Homira.osman@muscle.ca.

Join our Board of Directors

Muscular Dystrophy Canada is looking for volunteers to join our board of directors. If you have finance, risk management, human resources, marketing, not-for-profit management, or legal experience it would be an asset. Additionally, we are looking for people with lived experiences with neuromuscular disorders from different backgrounds from across Canada.

Time Commitment:

Each Director is elected to fill a two-year term
Each Director is expected to regularly attend four to six board meetings per year, with the opportunity to join a sub-committee (Governance and Mandate Committee or Finance, Audit and Risk Management Committee)
Meetings are scheduled from 7:00pm to 9:00pm Eastern Time with approximately two hours of preparation required prior to the meeting

Expectations/Responsibilities

Directors will work to enhance Muscular Dystrophy Canada’s standing by actively promoting our mission and accomplishments to members of the public.
Directors will support fundraising activities at MDC wherever possible including; through their own personally meaningful contributions, reaching out to personal contacts for partnerships and/or donations and participation in the stewardship of donors.
Directors may be asked to serve in an executive role (Chair, Vice-Chair, Treasurer or Secretary,) or join a subcommittee of the board as needed (Governance and Mandate Committee or Finance, Audit and Risk Management Committee).
Directors in consultation with our executive leadership team, are expected to participate in annual planning to ensure financial and operational goals of the organization are set.
Directors are to provide proper financial oversight by reviewing financial statements and key performance indicators (KPIs), as well as ensuring proper financial controls are in place.
Directors are expected to use due diligence and promptness in reviewing and approving financial and legal documents, as well as board meeting documents and responding to emails and inquiries.
Directors are expected to act with the highest degree of integrity and ethical behaviours.

Muscular Dystrophy Canada is committed to diversity, equity and inclusion, especially for persons with disabilities. We support equal opportunity and advancement in accordance with competence for all employees and volunteers.

If you are interested in being considered for our board of directors, please contact Benjamin Dixon at benjamin.dixon@muscle.ca.

NMD4C and MDC Launch Postdoctoral Fellowship Funding Competition

Together, Muscular Dystrophy Canada and the Neuromuscular Disease Network for Canada are excited to announce the launch of a funding competition to award two postdoctoral fellowships in neuromuscular disease (NMD) research!

The fellowship provides salary support for early-career scientists to conduct postdoctoral studies in a laboratory focused on research of NMDs, with successful applicants being eligible to receive up to $40 000 in funding over a one-year period. The fellowship will allow early-career scientists to pursue NMD-related research that will impact the understanding the biological causes and/or new clinical treatment of NMDs while also supporting the recipients to pursue future independent research careers in the field of NMDs.

Notice of 2021 Annual General Meeting of Members

NOTICE IS HEREBY GIVEN that the Annual General Meeting of the Members of Muscular Dystrophy Canada will be held via Zoom.

on Friday, September 24, 2021 at 2:15pm Eastern Standard Time for the following purposes:

Receiving the following reports:
- Chair of the Board and the CEO;
- Treasurer; and
- Independent Auditor, together with the audited financial statements for the year ended March 31, 2021.
Electing the Board of Directors
Appointing the Auditors
Other business as may properly be brought before the meeting.

Members of the public are welcome to attend the meeting. If you would like to attend, please contact Benjamin Dixon at benjamin.dixon@muscle.ca or 800-567-2873 to obtain the videoconference details.

Dave Ferguson
Secretary of the Board of Directors
Cowichan Bay, BC

National collaboration awards funding for newborn screening in spinal muscular atrophy (SMA)

SMA is the leading cause of genetic infant death.
Early diagnosis is imperative to halt irreversible motor neuron loss and disease progression.
More than $700,000 awarded to projects across three provinces.

Toronto, ON, July 21, 2021 – Muscular Dystrophy Canada (MDC) today announced funding for newborn screening projects focused on spinal muscular atrophy from Alberta, Manitoba and Quebec. These are the first applicants to be awarded funds as part of a collaboration with Novartis Pharmaceuticals Canada Inc. (Novartis). This multi-year initiative will see up to $2 million invested, as well as ongoing support for the generation of critical evidence and best-practices to help make newborn screening for SMA a national reality.

“In a neuromuscular disorder like SMA, where time is of the essence, early diagnosis and prompt access to treatments are critical to achieving the best possible outcomes. Unfortunately, this is not the reality for most Canadian children born with SMA,” said Stacey Lintern, CEO, Muscular Dystrophy Canada. “With today’s announcement and the unparalleled commitment of government leaders, Novartis, the SMA community and MDC’s dedicated Board of Directors, Fire Fighters, clients, donors and supporters, we are one step closer to ensuring all Canadian newborns are screened for SMA.”

Newborn screening is a test done for babies shortly after birth to look for treatable diseases that usually show no symptoms in the newborn period. While Ontario is currently the only province to screen for SMA, MDC’s goal is to have the disorder added to all provincial newborn screening panels across Canada.

“As the mother of an infant with SMA who was not screened at birth, I know first-hand the devastating implications of delayed diagnosis,” said Laura Van Doormaal, Newborn Screening Advisory Committee member. “Today’s announcement means that one day soon, no matter where they live all families will have the opportunity to make critical healthcare decisions and realize their child’s full developmental potential from the very beginning.”

In order to ensure an evidence-based approach to funding, MDC and Novartis commissioned a readiness assessment to evaluate the feasibility of adding SMA to all provincial and territorial screening panels. Proposals were then evaluated by an independent international peer review committee under MDC’s guidance. Funded projects include pilot programs and the mapping of post-diagnosis referral pathways. Further details on the needs and readiness assessment, project selection, peer review and community advisory committees can be found here: muscle.ca/newborn-screening

“Our collaboration with Muscular Dystrophy Canada truly exemplifies the Novartis purpose of improving and extending patients’ lives,” said Andrea Marazzi Country Head, Novartis Pharmaceuticals Canada. “We’re honoured to partner on work that will bring lasting impact to the SMA community and healthcare systems across Canada. Time to diagnosis is crucial in SMA and we applaud the first round of funding recipients and all engaged provinces for their commitment to prioritize screening at birth.”

MDC will continue to work with British Columbia, Saskatchewan and the Maritimes to accelerate newborn screening for SMA coast-to-coast. Future phases of the national collaboration will include additional project funding, evaluation of projects and knowledge transfer, and exchange with stakeholders to ensure policy adoption across all provinces and territories.

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.

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For more information:

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

Call for Project Proposals: Newborn Screening for Spinal Muscular Atrophy in Canada

Today, we are excited to be launching Phase 2 of our work with a call for funding proposals for local NBS for SMA initiatives. It is anticipated that these projects will focus on the setting up of laboratories for testing and/or ensuring efficient uptake of the policy itself. An independent selection committee will review applications in the coming weeks and we expect to announce the first round of funding at the end of June.

LEARN MORE

Progress on national newborn screening for SMA

To our community,

For many neuromuscular disorders, early diagnosis and prompt access to treatments are integral to achieving positive outcomes. Unfortunately, this is not the reality for most Canadian children born with spinal muscular atrophy (SMA). When time equals neurons, diagnosis at birth is vital. Newborn screening (NBS) gives families the opportunity to make important healthcare decisions from the very beginning and gives every child a chance to realize their developmental potential.

Screening for SMA can detect approximately 97% of cases. Yet Ontario is currently the only province with SMA on their screening panel.

In December 2020, we embarked on a multi-year collaboration with Novartis Pharmaceuticals Canada Inc. to make NBS for SMA a national reality. We recently completed Phase 1 of the project, which assessed the feasibility of including SMA into existing NBS programs and created a readiness assessment plan for implementing a standardized SMA screening program in Canada. Provincial readiness was assessed on the following key factors:

Existing infrastructure to conduct specialized screening
Accessible treatment
Favourable environment to add SMA to NBS panel
High readiness among clinical stakeholders to support SMA NBS program
Existing infrastructure in-province to confirm diagnosis of SMA through genetic testing
Low cost for adding SMA to the NBS

Despite immediate challenges, the assessment highlighted a strong consensus on the value of implementing SMA in NBS programs across Canada and a shared understanding of the urgency.

We look forward to receiving proposals from medical and laboratory experts across the country. But our work will not stop there. We will continue to engage with policy and decision makers in all jurisdictions to secure NBS for SMA for every newborn.

With support from the SMA community from coast to coast, we are standing up for Canadian babies. Join us in the call to #screenforsma.

Stacey Lintern
Chief Executive Officer

MDC funds seven new projects to propel research forward during challenging times

Toronto, Ontario – Muscular Dystrophy Canada (MDC) is proud to announce an investment of $400,000 to fund seven new research projects in 2021 through the MDC Neuromuscular Disorder (NMD) Research Grant Competition, a dedicated Canadian source of funding for neuromuscular research. MDC will also invest in an international, collaborative, $1.15 million, research project through the European Joint Programme on Rare Diseases (EJP RD).

“Despite an unprecedented time, during which many like-minded organizations and agencies are not in a position to offer research funds in Canada, MDC is able to demonstrate its ongoing commitment to discovering cures,” stated Stacey Lintern, CEO, Muscular Dystrophy Canada. She added, “while others redirected their research funds towards the coronavirus, and this was much-needed, MDC recognized the need to maintain momentum, and ensure the incredible advances currently taking place in research are accelerated.”

In addition to the seven new projects being funded, MDC will continue its support of 12 active research studies from the previous year.

“MDC grant recipients are selected by medical and scientific professionals, as well as Canadians with lived experience. Research proposals are judged based on scientific merit, and the potential to translate findings into initiatives that will positively impact the NMD community,” said Dr. Daria Wojtal, Director of Research, Muscular Dystrophy Canada.

MDC relies on the generosity of donors to fund life-changing research.

Dr. Rashmi Kothary, Deputy Scientific Director and Senior Scientist at the Ottawa Hospital Research Institute and 2019-2020 MDC research grant recipient, said “to the families and donors, I would have to say a huge thank you. From my first day as a researcher, getting my first grant from MDC, you were there. Thank you for sticking by the research community for many, many years. Hopefully we can pay back the trust that you put in us.”

MDC is honoured to fund the exceptional and bright researchers, clinicians and academics who will lead these seven new projects taking place in hospitals and universities across Canada, and around the world.

The 2020-2021 research grant recipients are:

Dr. Alex Parker
A Pharmacogenetic Pipeline for Charcot-Marie-Tooth Disease
Dr. Colin Crist
Promoting Muscle Repair by Pharmacological Inhibition of eIF2a Dephosphorylation
Dr. Mohamed Chahine
Human iPSC-derived Neurons as a Model of Congenital Myotonic Dystrophy Type 1
Dr. Nadine Wiper-Bergeron
Improving Myoblast Transplantation Outcomes via Pharmacological Reprogramming
Dr. Nicolas Dumont
Targeting Defective Stem Cells in a Preclinical Model of Myotonic Dystrophy Type 1
Dr. Rima Al-Awar (EJPRD/CIHR Co-funded)
Safety and Efficacy of a Possible Epigenetic Therapy for Facioscapulohumeral Muscular Dystrophy
Dr. Toshifumi Yokota
Enhancing the Efficacy of Antisense Oligonucleotide Therapy for Facioscapulohumeral Muscular Dystrophy

“Congratulations to the 2020-2021 grant recipients. MDC is committed to supporting opportunities that fulfill our commitment to invest in research that leads to information on diagnosis, treatment and health management of neuromuscular disorders; and, we’re confident that the research funded this year will bring us closer to cures for all children, youth, adults and families affected by neuromuscular disorders,” added Lintern.

DISCOVER THE PROJECTS WE SUPPORT

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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 explore our website 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