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.

 

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Reduce the timing between doses for patients with neuromuscular diseases

Jurisdictions across the country have been following National Advisory Committee on Immunization (NACI) recommendation to put off second doses and prolong the time between the first and second doses of two-shot COVID-19 vaccines to up to four months. However, persons with neuromuscular disorders may have respiratory involvement, require immunotherapy and are particularly vulnerable to severe and life-threatening effects from contracting COVID-19 infection. Partial protection for some of the most vulnerable and high-risk patients in the country from this devastating disease is not acceptable, particularly as the virus and variants surge across the country. Together with neurologists, the Neuromuscular Disease Network for Canada, Jesse’s Journey and GBS-CIDP Canada, MDC submitted a letter to Health Minister Patty Hajdu, Dr. Quach-Thanh, Chair of NACI and each provincial health minister urging them to further nuance the list of those who should be prioritized for their second dose by including adults affected by neuromuscular disease.

If need any support accessing your first and/or second dose of the COVID-19 vaccine, please feel free to contact advocacy@muscle.ca.

Open letter to Health Minister Patty Hadju
Open letter to NACI

MDC Change Agents 

As we celebrate National Volunteer Week, we are happy to share that the following members for MDC’s Advocacy Advisory Committee have been selected:

Catherine Boivin
Quebec
Profession: Digital Technologies Specialist and Consultant
Connection to MDC: Adult affected by a NMD

Alfred Breton-Pare
Quebec
Profession: Senior CRA; Project Manager; Clinical Research Manager
Connection to MDC: Parent of a child affected by a NMD, MDC Board Member

Emily Hong-Wing Chan, BSc (Hons), MSW (Cand.)
Ontario
Profession: Social Worker
Connection to MDC: Adult affected by a NMD

Dr. Sue Dojeiji MD, Med, FRCPC
Ontario
Profession: Physiatrist-in-Chief, Clinical educator; Neuromuscular Researcher
Connection to MDC: NMD Clinician-Scientist

Susan Jahnke
British Columbia
Profession: Business Owner & Website Manager
Connection to MDC: Adult affected by a NMD

Corrine Kagan, BA (Hons)
Ontario
Profession: Past Senior Program Director (ABI)
Connection to MDC: Adult affected by a NMD

Dr. Colin Kovacs, MD, Med, FRCPC
Ontario
Profession: Family Medicine/HIV Primary Care Physician; Assistant Professor in Internal Medicine at U of T
Connection to MDC: Adult affected by a NMD, Member of NMD4C

Ken Kramer, Q.C.
British Columbia
Profession: Principal & Senior Associate Counsel at KMK Law
Connection to MDC: Adult affected by a NMD

Louis Joseph Lauziere
Ontario
Profession: Past Senior Policy Advisor in Dept of Justice at Gov of Saskatchewan
Connection to MDC: Adult affected by a NMD

Michael Low, JD
Alberta
Profession: Litigation Associate at Fasken Martineau DuMoulin LLP
Connection to MDC: Parent of a child affected by a NMD

Stefanie Marinich-Lee, LLB.
Ontario
Profession: Lawyer, Corporate HR
Connection to MDC: Adult affected by a NMD

Erin Novakowski
Alberta
Profession: Undergraduate student at U of Alberta; Disability Social Media manager; Writer and Contributor for Accessible Housing Canada
Connection to MDC: Adult affected by a NMD

Thu Parmar, B.Sc. Pharm
Alberta
Profession: Clinical Pharmacist; Managed public drug programs at Alberta MoH; Senior Manager of Government Relations & Public Policy at Hoffmann-La Roche Limited (Roche Canada)
Connection to MDC: NMD Industry Partner

Jalee Pelissier
Ontario
Profession: PT/OT Aide
Connection to MDC: Adult affected by a NMD

Kara Reid, BSc OT
New Brunswick
Profession: Occupational Therapist
Connection to MDC: Member of Neuromuscular Clinic, MDC Board Member

Dr. Kerri Schellenberg  MD, MMedEd, FRCPC, CSCN (EMG)
Saskatchewan
Profession: Neurologist and Medical Director ALS Clinic; Neuromuscular researcher.
Connection to MDC: NMD Clinician-Scientist

Erica Vella, BA
Ontario
Profession: Digital Broadcast Journalist with Global News Toronto
Connection to MDC: Family member of an adult affected by NMD

Fatima Vido-Vecchio, B.Sc. PT
British Columbia
Profession: Physiotherapist
Connection to MDC: Member of Neuromuscular Clinic

Elaine Whitmore
Ontario
Profession: Past CEO of John McGivney Children’s Centre
Connection to MDC: Friend of persons affected by NMD; Advocate

We are thankful to all who applied and applaud our new members for coming forward to help us work together towards inclusive policies that reflects and respects Canada’s diversity, values the neuromuscular experience and are accessibility-focused.

We look forward to influencing positive change together! If you have any questions about advocacy, please email advocacy@muscle.ca

Researchers from across Canada join The Tenaquip Foundation’s Walk4MD to raise awareness and funding for neuromuscular disorder community

FOR IMMEDIATE RELEASE April 15, 2021

Toronto, Ontario – For the second year in a row, Muscular Dystrophy Canada (MDC) is bringing the neuromuscular disorder (NMD) community together virtually for its annual Walk4MD, which raises much needed funds to support Canadians affected – and this year researchers are joining the Walk4MD family.

On June 19 at 3 p.m. EST, thousands of Canadians including six prominent NMD researchers across the country will come together, virtually, to participate in The Tenaquip Foundation’s Walk for Muscular Dystrophy (Walk4MD).

Dr. Reshma Amin from the Hospital for Sick Children in Toronto is excited to be one of the Walk4MD Research Champions. “I look after so many amazing children with neuromuscular disease and their families. They deserve to be celebrated and what better way to do so than in partnership with MDC through the Walk4MD.”

“Important advancements are taking place in research right now, and we want to ensure that these advancements continue, and result in better treatments and ultimately a cure for neuromuscular disorders. We feel very fortunate to have the support of Dr. Reshma Amin, MD; Dr. Bernard Brais, MD, PhD; Dr. Nicolas Chrestian, MD; Dr. Robin Parks PhD; Dr. Gerald Pfeffer, MD, PhD; and Dr. Martine Tetreault, PhD and look forward to our NMD community getting to know these incredible individuals better,” said Stacey Lintern, CEO, Muscular Dystrophy Canada.

She added, “I’m also pleased to share that The Tenaquip Foundation is joining us again as title sponsor, and Global has returned as media sponsor for the second year in a row. Like us, they are committed to raising awareness and funds for the Canadian NMD community.”

The Tenaquip Foundation’s Walk4MD is a signature fundraising event for MDC that raises critical funds to support Canadians impacted by neuromuscular disorders. This year, due to the ongoing COVID-19 pandemic, the funds raised are more important than ever.

“There is still urgency to raise funds to meet the increased needs of our clients during this time,” says Lintern. “Like last year, we’ve had to cancel the majority of our fundraising events for the safety and well-being of our team and the NMD community. That unfortunately, has resulted in a 60 per cent decrease in revenue in our first quarter last year.”

The goal of The Tenaquip Foundation’s Walk4MD this year is to raise $870,000. These funds will be invested in services and programs, like equipment, and ensure MDC can meet the unique needs of the neuromuscular community while continuing to invest in research for better health outcomes for individuals impacted by neuromuscular disorders.

To learn more about The Tenaquip Foundation’s Walk4MD or to register for this exciting event, please visit walk4MD.ca

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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.

MEDIA CONTACT

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

MDC’s Response to the CADTH and INESSS Recommendations for Onasemnogene abeparvovec: A Call for Newborn Screening

At the end of 2020, Health Canada approved Onasemnogene abeparvovec (Zolgensma®) for the treatment of pediatric patients with 5q Spinal Muscular Atrophy (SMA) with biallelic mutations in the SMN1 gene and three or fewer copies of SMN2 gene, or infantile-onset SMA, bringing renewed hope to Canadian families affected by SMA. Onasemnogene abeparvovec is the second treatment approved by Health Canada for SMA and the first gene therapy for SMA. The broad indication as part of Health Canada approval was followed up with recommendations from the Institut national d’excellence en santé et en services sociaux (INESSS) and most recently, the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committees. The recommendations were formulated following a comprehensive evidence-based review of the medication's efficacy or effectiveness and safety and an assessment of its cost-effectiveness. The CADTH recommendations note that Onasemnogene abeparvovec should be for patients who are: symptomatic or pre-symptomatic with one to three copies of the survival motor neuron gene; 6 months of age or younger; not currently requiring permanent feeding or ventilatory support (either invasive or non-invasive).

Based on the patient input we put forward to CADTH and INESSS, we know SMA contributes to a loss of independence, increased load on families, difficulty breathing, swallowing and loss of mobility and can have a negative impact on mental and emotional well-being. Since Health Canada’s approval of treatments for SMA, families have been anxiously approaching age limitations and tirelessly advocating for access. While we commend INESSS and CADTH for their thorough class reviews, we are disappointed to see Onasemnogene abeparvovec is not recommended for those over the age of 6 months and a case-by-case review mechanism has not been suggested by CADTH. This emphasizes the need for a comprehensive Rare Disease Strategy and a process/mechanism to address the evidence gap for older infants and children. We thank the clinical experts for contributing to the reviews and we are in full agreement with the clinical experts that “earliest possible initiation of therapy” is important when it comes to SMA because it is associated with irreversible loss of motor neurons and motor nerves. This means in order to best benefit from the treatments currently approved and available in Canada, a diagnosis should be available as early as possible. Thankfully, this is the case in Ontario – newborn screening for SMA is part of the infant screening panel at birth since 2020. However, for anyone outside of Ontario, a diagnosis prior to 6 months of age is not always achieved. The recent INESSS and CADTH recommendations highlight the critical need for newborn screening, especially in the presence of approved treatments, and strongly provides support for MDC’s current initiative to implement nation-wide newborn screening for SMA.

We acknowledge that the recent approval of another treatment for SMA and related drug review recommendations has taken us another significant step towards early diagnosis and treatment choice. MDC is committed to continually work with individuals affected by SMA and their families on an individualized basis to provide holistic supports, resources for making evidence-informed choices and supporting individual advocacy activities.

We are looking forward to continued research and developments that can demonstrate the meaningful benefits of early diagnosis and early access to treatments for health outcomes and health-related quality of life for Canadians affected by SMA.

Sincerely,
Stacey Lintern
Chief Executive Officer
Muscular Dystrophy Canada



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Muscular Dystrophy Canada Receives National Accreditation

March 3, 2021 – Muscular Dystrophy Canada (MDC) has been awarded accreditation in Imagine Canada’s Standards Program for excellence in non-profit accountability, transparency, governance and fundraising. Created “by the sector for the sector”, the Imagine Canada Standards Program is the only national accreditation program that is designed for all Canadian charities and non-profits.

To earn the accreditation, organizations must demonstrate excellence in five fundamental areas: board governance; financial accountability and transparency; fundraising; staff management; and volunteer involvement. Accreditation is a sought-after hallmark of excellence within the sector for funders, donors, sponsors and supporters alike.

Organizations receiving the accreditation are awarded a Trustmark to signal credibility and build confidence with donors.

“The Imagine Canada Standards Program accreditation demonstrates MDC’s commitment to our donors, supporters, all Canadians who are impacted by neuromuscular disorders, and to the work that we do,” said Donna Nixon, Chair, Board of Directors, Muscular Dystrophy Canada.

Nixon added, “We’ve always held to these standards, but by completing this comprehensive application and peer-review process we’ve been able to refine and evolve some of our practices to further strengthen the organization. My fellow directors, the leadership team and staff at MDC were all instrumental in making this accreditation possible. I thank them for their hard work and commitment through the application process, and for ensuring that MDC is upholding Imagine Canada’s standards for excellence and leadership in the non-profit sector each and every day.”

“Earning the accreditation, especially in these changing times, shows remarkable resilience, and a strong commitment to innovation and excellence. We commend the latest well-deserving recipients of the Trustmark for achieving Imagine Canada’s Standards accreditation, and demonstrating outstanding adherence to sound governance,” said Bruce MacDonald, President and CEO of Imagine Canada.

To learn more, visit imaginecanada.ca.

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ABOUT MUSCULAR DYSTROPHY CANADA (MDC)

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. Learn more about MDC online, or call our toll-free number at 1-800-567-2873.

ABOUT THE IMAGINE CANDA STANDARDS PROGRAM

The goals of Imagine Canada’s Standards Program are to increase organizational excellence and transparency of charities and nonprofits, to build resilience and to strengthen public confidence in individual organizations and the sector as a whole. To earn the accreditation, organizations must meet 73 standards in financial management and accountability, fundraising practise, board governance, staff management, and volunteer management.

MEDIA CONTACT INFORMATION:

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

Muscular Dystrophy Canada calls on provincial governments to prioritize persons affected by neuromuscular disorders for COVID-19 vaccine

February 23, 2021 – Many people living with neuromuscular disorders (NMD) are at greater risk of developing serious symptoms and complications as a result of COVID-19 due to their often pre-existing cardiac and respiratory issues and chronic comorbidities. As the voice of the NMD community in Canada, Muscular Dystrophy Canada (MDC) is calling on governments to prioritize people living with hereditary and acquired neuromuscular disorders in their vaccination roll-out.

Specifically, MDC is asking that the following groups of people with neuromuscular disorders be prioritized: adults requiring multiple caregivers or complex ongoing support in the home, community or institutional setting; the caregivers or home care workers who provide ongoing support to children and adults with neuromuscular disorders; adults with severe or unstable respiratory issues; adults with reliance on home ventilation; adults with clinically relevant impairment of heart function; and, adults receiving immunosuppressants.

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Your Impact on the Neuromuscular Community in 2020 was Profound

Welcome to 2021. I think it’s safe to say we’re all looking forward to the fresh start and renewed possibilities that this year will bring.

I want to take this opportunity to thank you for your support of the Canadian neuromuscular community in 2020. It was a challenging year for all, but your support directly impacted thousands of families and individuals across the country. Your donations funded access to treatments, programs and services and ground-breaking research.

For instance, you’re helping ensure early screening and treatment is available regardless of where you live. Thanks to the support of donors like you, babies and families across the country are a step closer to early diagnosis and treatment. We’re very excited to embark on a nation-wide newborn screening collaboration in 2021 to fuel the goal of making newborn screening for spinal muscular atrophy (SMA) a reality.

Also because of you, individuals affected by neuromuscular disorders stayed connected with their community, and were provided knowledgeable advice from experts in the health and medical fields during the pandemic. Your support allowed us to offer network meetings and educational webinars, virtually, so individuals could participate from the safety of their homes.

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Donors, like you, also played a vital role in keeping families together over the holidays. A research project you funded, A Virtual Transition Intervention for Children and Adults Transitioning To Home Ventilation, brought complex ventilator support into homes virtually. Individuals requiring ventilators to help with breathing make frequent visits to hospitals for specialized care and to ensure their equipment is working properly. This year, given these individuals are at a high risk for respiratory illness it was more important than ever that they stay at home with close family and friends. Thanks to your support of this research project, a virtual support program was piloted to bring medical experts into the homes of these individuals, reducing the need for hospital visits and possible exposure to COVID.

When we work together, we can truly accomplish incredible things.

That’s why I’m reaching out to you today. Will you renew your commitment to the neuromuscular community in 2021 by making a donation to MDC? Your gift will mean more investments in research, life-changing partnerships and continued support for individuals and families regardless of where they live.

The beginning of a new year is also the perfect time to become a monthly donor. Monthly donations support long-term projects and initiatives. They keep vital programs and services going all year long and ensure that critical advocacy efforts can continue to make positive changes for our community. The pandemic has underscored the vital importance of each and every donor and a sustainable monthly gift provides certainty for our ongoing work.

I know with your renewed support, we can continue to make meaningful changes, invest in research and form valuable partnerships that support Canadians affected by neuromuscular disorders.

Partnerships, such as the one with Thames Valley Neuromuscular Clinic where a pilot program is helping pediatric patients and their families make informed decisions throughout the care journey to alleviate some of the fear and stress these parents often experience. Your donations are also behind our Systems Navigation program, which helps Canadians with all areas of their non-medical needs. This past year, your gifts ensured MDC was able to continue funding critical equipment for individuals impacted by a neuromuscular disorder despite a drastic reduction in fundraising revenue.

MDC is the only national charity that supports Canadians with a wide range of neuromuscular disorders across the country, but we can’t do it without you. The impact you are having on the neuromuscular community is profound.

I hope we can count on you to renew your support today by making a donation.

Again, thank you for your support in 2020 and I look forward to working with you to continue changing lives in 2021. Because, like us, I know you too are ignited by passion and fuelled by hope.

Warm regards,
Stacey Lintern
CEO, Muscular Dystrophy Canada

P.S. Our new year appeal is one of our most important. Donations from supportive Canadians like you are put to work right away to support families and to fund research. While hope is renewed with the recent COVID vaccine, charities like ours continue to be impacted by its effect on our traditional fundraising activities and the economic realities for so many. We hope you can continue your support for 2021.