NMD4C and MDC Launch Postdoctoral Fellowship Funding Competition

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.

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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

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

COVID – 19: What you should know about this novel coronavirus

Given the recent updates on COVID-19, Muscular Dystrophy Canada has implemented a work from home policy, therefore, effective immediately all MDC offices are closed.

Our staff remain available via email and telephone and will continue to serve our clients, volunteers, donors and other key stakeholders.

During this difficult time, we remind everyone that social distancing is a critical component of reducing the potential harm.

In the coming days, MDC will continue to provide updates as we strive to implement new online platforms and solutions to support our community.

We recommend that everyone continue to be informed by credible sources like the World Health Organization, the Public Health Agency of Canada and provincial health agencies.

Thank you for your continued support and cooperation.

Barbara Stead-Coyle
CEO

COVID-19, is a coronavirus, that is known to cause respiratory infections in individuals who contract the virus. Symptoms include dry cough, fever, tiredness and difficulty breathing, and may take up to 14 days to appear after exposure. Since people with neuromuscular disorders already experience respiratory challenges, it is understandable that you would have concerns and questions about COVID-19.

How to prevent infections

The steps outlined below are good practice, at all times, to inhibit the spread of viruses:
  • Wash your hands thoroughly and regularly with soap and water.
  • Sneeze and cough into your sleeve; when using tissues, immediately put them in the trash and wash your hands.
  • Avoid touching your eyes, nose or mouth.
  • Regularly clean commonly used surfaces and devices you touch.
  • Avoid crowded spaces and close contact with anyone that has a fever or cough.

How you can prepare

You should always have a plan in place should you, or a loved one, become ill. You should have:
  • A list containing the names of your doctors, clinic, pharmacy and insurance company along with contact information.
  • A list of all your medications and the doses.
  • Enough medication for one to two weeks in case you cannot get to the pharmacy to refill your prescriptions.
  • Extra supplies like, hand sanitizers and soap to wash your hands.
For more information please visit the Government of Canada’s website.

How to avoid disruptions to care services for individuals with neuromuscular disorders

  • Screen: Have a sign posted at the entrance of your home for your caregivers and attendants to self-screen and review the precautionary measures they can take while in your home.
  • Greetings: Have your caregivers and attendants say hello without touching (e.g., a wave hello)
  • Wash Hands: Have your caregivers and attendants wash their hands for at least 20 seconds with soap carefully and repeatedly (throughout the day).
  • Sanitize: Have your caregivers and attendants use hand sanitizer (at least 60% alcohol-based) when they arrive at your home and each time prior to touching or feeding you.
  • Disinfect: Have your caregivers and attendants clean, sanitize and disinfect the surfaces that are touched in your and use disinfecting wipes on items that are frequently touched (e.g., cell phones, doorknobs, your wheelchair controls, lifting device controls, and remote controls). Make this convenient by having wipes near the items that should be regularly cleaned.
  • Back Up: If your attendant becomes sick, ask them to seek medical care. Ensure you have sufficient back up attendants in case your caregivers and regular attendants cannot work; your attendants may not be able to work because either they get sick or they need to take care of a family member who is. You will still need assistance, so make sure you have someone who can provide it.

What to do if you think you might have the infection

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

Major Improvement in CRISPR Technology Could Fix Genetic Spelling Mistakes: Implications for Neuromuscular Disorders?

CRISPR-Cas9 technology, a gene editing tool, has been significantly upgraded with a new feature called prime editing. This enhancement addresses two limitations of CRISPR technology by: (1) allowing the change of a gene’s spelling into a predicted sequence without requiring a cell to divide and (2) without needing to cut both strands of the DNA double helix, which reduces the risk of making unintentional changes. The study published in Nature used prime editing on human cells to correct the genetic spelling mistakes associated with Tay-Sacks and Sickle-Cell Anemia. Compared to traditional CRISPR technology, this study showed that the prime editing feature made CRISPR more precise, efficient and highly versatile.

CRISPR-Prime editing holds promise for the treatment of neuromuscular disorders (NMDs) as it allows genetic repairs in non-dividing cells like neurons and muscle cells and could potentially be used on a subset of NMDs caused by small genetic spelling mistakes like point mutations, small insertions or deletions. But in its current form, up to 80 letter changes were edited and so this upgrade would not work for other types of NMDs: Charcot-Marie-Tooth 1A and Myotonic muscular dystrophies as well as some cases of Duchenne, and Becker Muscular Dystrophies are caused by large thousand or even hundred thousand letter deletions, duplications, inversions and repeat expansions. As this technique moves us one step closer to correcting genetic mutations that cause human genetic diseases, there is a need to continue research to develop safe and effective treatment for the wide range of specific disease-causing spelling mistakes associated with NMDs

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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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