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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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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MDC presents CADTH With GMG Patient Submission

MDC pleased to provide CADTH with a patient submission from our generalized Myasthenia Gravis (GMG) community in support of Soliris. MDC remains committed to ensuring that neuromuscular clients have access to the latest treatments that offer positive health outcomes.

Download the patient submission

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

Treating Duchenne Muscular Dystrophy Focus of New Research Partnership

For Immediate Release – April 4, 2019

Toronto, Ontario – Muscular Dystrophy Canada (MDC) and The Foundation for Gene & Cell Therapy (Jesse’s Journey) are joining forces to accelerate ground-breaking research focused on new treatments for Duchenne muscular dystrophy to the sum of $600,000.

Through this partnership, MDC will provide $300,000 with Jesse’s Journey matching the commitment. Two research projects will be funded:

  • Dr. Anthony Gramolini: Non-viral, immune-modulatory nanoparticles for delivery of CRISPR/Cas9 as a treatment intervention for Duchenne Muscular Dystrophy.
  • Dr. Michael A. Rudnicki: Exosomal Delivery of Wnt7a for treating Duchenne Muscular Dystrophy.

“For anyone impacted by a neuromuscular disorder, research offers hope that better treatments will be uncovered, and ultimately, a cure. Both of these research projects show very promising initial results and we are thrilled that this investment will help move the research forward,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We are thrilled to be working with Jesse’s Journey to fund this promising research and can’t wait to see what positive impacts it has on our clients and families.”

Barbara Stead-Coyle
CEO, Muscular Dystrophy Canada

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Federal Government Commits to National Strategy to Improve Access to Rare Disease Drugs

Toronto, Ontario – Muscular Dystrophy Canada commends the Government of Canada for committing $35 million, in its 2019 budget, to develop a Canadian Drug Agency to oversee the development of a new national formulary of prescribed drugs and a strategy to provide support for Canadians with rare diseases.

A national strategy to alleviate the high-cost of drugs for rare diseases will ensure more Canadians are able to access effective, life-changing treatments.

“This is an important first-step towards ensuring individuals diagnosed with a rare disease, such as a neuromuscular disorder, are able to access treatment without cost as a barrier,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We call on all Federal parties to focus on this very real issue facing Canadians.”

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