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

Response to PMPRB Draft Guidelines Consultation

February 14, 2020

Dr. Mitchell Levine
Chairperson
Patented Medicines Prices Review Board
333 Laurier Avenue West, Suite 1400 Ottawa, Ontario K1P 1C1
Email: PMPRB.Consultations.CEPMB@pmprb-cepmb.gc.ca

Dear Dr. Levine:

Muscular Dystrophy Canada is thankful for the invitation to attend the PMPRB Consultation meeting in Ottawa on December 10, 2019 and for the opportunity to respond to the Draft Guidelines Consultation.

Muscular Dystrophy Canada leads Canada’s neuromuscular community as the central axis in a complex health services, policy, and information ecosystem that surrounds and support our core concern: Canadians with neuromuscular disorders. Muscular Dystrophy is the umbrella name used to cover the 160-plus known neuromuscular disorders today. While the disorders we represent are individually rare, they are collectively common, impacting more than 50,000 Canadians from coast to coast. These disorders weaken the body’s muscles, or the communication between the nerves and the muscles. The causes, symptoms, age of onset, severity and progression vary depending on the individual and the exact diagnosis. For many individuals, the disorder is fatal. Currently, there is no cure for these disorders. We believe that neuromuscular disorders will one day be cured (with treatments and therapies), and that our organization will be a positive contributor to that outcome by: investing in innovative research that has potential for real-world impact; providing programs and support services for those impacted by neuromuscular disorders, their caregivers and families, health professionals and researchers; and remaining committed to advocating for fair and patient-centric policies that improve the lives of all Canadians with neuromuscular disorders.

Currently, the range of treatments and therapies available to support impacted individuals can come with a heavy financial burden and a significant economic impact. As an organization, we are passionate in the belief that every Canadian with a neuromuscular disorder deserves to benefit from leading-edge discoveries in the research field in a timely manner and equal access to the best treatments. Muscular Dystrophy Canada together with members of its Medical and Scientific Advisory Committee are of the strong belief that, if PMPRB guidelines are enacted as proposed, the amendments may result in unfavorable consequences as described below:

Significant decreases in price will result in delays in manufacturers launching their product in Canada and this will have a negative impact on the overall length of time that it takes for Canadians to have access to new medicines in Canada, if at all. We are seriously concerned that Canadians with neuromuscular disorders will, in fact, see a decline in innovative medicines access. Members of our Medical and Scientific Committee Dr. James Dowling (Hospital for Sick Children, Toronto), Dr. Ronald D. Cohn (Hospital for Sick Children, Toronto), and Dr. Craig Campbell (Children’s Hospital London Health Sciences Centre, London) together published an article in April 2018 in the Journal of Medical Genetics that emphasizes: “many exciting treatment approaches are currently in clinical trial, and several have achieved conditional or full market approval in various regions of the world. Many other treatments are in the pipeline, and we predict that over the next decade meaningful therapies will become widespread across the neuromuscular disease spectrum.”

In addition to compromising timely access to new therapies for patients with neuromuscular disorders, Muscular Dystrophy Canada is profoundly concerned about the impact the pricing changes will have on the health research infrastructure of Canada. Although PMPRB confidently notes that pricing is not a significant determinant to bringing clinical research to Canada, the Life Sciences Ontario (2019) survey says otherwise. In that survey, 91% of pharmaceutical executives said the changes would have a negative effect on clinical research in Canada, with 44% saying the negative effect would be “significant.” There will be significant adverse impact of the proposed changes on research incentives and investments, which is important as we are on the verge of life-changing therapies and treatments for individuals with neuromuscular disorders.

While we support the efforts to lower the costs of prescription drugs for Canadians, we strongly believe that this must be done in a way that ensures timely access by Canadians to new medicines and to clinical studies of new medicines. Our recommendations are in line with those proposed by Health Charities Coalition Canada (HCCC) and we strongly echo the discussion outlined in their thorough and carefully well thought-out submission. We suggest you carefully review our submissions and take into account these four key recommendations:

Recommendation #1:

That the PMPRB undertake a stepwise approach to its proposed changes by initially enacting only the changes to the comparator countries. Once the impact of this change is fully understood and if the objective of lowering Canadian prices sufficiently has not been met, then other new elements could be considered.

Recommendation #2:

That a multi-stakeholder dialogue be established to evaluate the impact of the changes on availability of medicines and specifically to inform any decision on whether and how to implement the use of the new economic criteria.

Recommendation #3:

That the Federal Government require PMPRB to hire a third party to conduct a formal assessment of the potential and real-time impacts of the reforms on research investment and activity in Canada (including clinical trials).

Recommendation #4:

That the Federal Government require that PMPRB, along with other appropriate agencies, immediately establish a formal mechanism for meaningfully and continuously engaging patient representatives in its decision-making and processes to ensure patient voice, choice and representation.

Thank you for the opportunity to review the guidelines and provide comments. We remain committed to working with the Federal Government and the broader stakeholder community in defining an implementation and evaluation process that will address both cost savings and access

to innovation that will optimally serve the needs of individuals with neuromuscular disorders in Canada. We wish our feedback will be taken into consideration as you prepare the final guidelines and we would be happy to answer any questions you might have.

Kind Regards,
Muscular Dystrophy Canada

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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Patients Living with Spinal Muscular Atrophy (SMA) in the Province of Saskatchewan Gain Access to Spinraza™

April 22, 2019

Dear members of the SMA community,

In response to the requests received by patients living with Spinal Muscular Atrophy (SMA), Biogen Canada would like to provide this latest progress regarding coverage of SPINRAZA™ (nusinersen) in the province of Saskatchewan.

Biogen Canada is delighted to inform you that on April 12, 2019, the Government of Saskatchewan informed treating physicians in their province that patients living with Spinal Muscular Atrophy (SMA) have access to SPINRAZA™. The Saskatchewan Ministry of Health has made the decision to expand coverage of SPINRAZA™ to include the following, in addition to existing Type I patients:

  • Patients who are pre-symptomatic with two or three copies of the SMN2 gene;
  • Patients up to age 18, with symptom onset after six months of age and who have never achieved the ability to walk independently;
  • Patients that may have achieved the ability to walk independently (type III) as well as type II and type III patients over the age of 18 are encouraged to talk to their treating physician to apply for a case by case coverage.
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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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