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Alberta fourth province to expand access to SPINRAZA™ for patients impacted with Spinal Muscular Atrophy

For Immediate release – June 25, 2019
Toronto, Alberta

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 after 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.

SMA causes major problems with walking, muscle strength, fine motor skills, and the basic physical functions of breathing, swallowing, and feeding.  SPINRAZA™ is a medication that has been described by patients as a “miracle”. Demonstrated improvements include stronger breathing and speech, increased strength and energy, and the ability to perform new actions such a rolling over and sitting or standing up without assistance.

This announcement follows closely on the heels of Ontario’s approval of expanded access, and comes after a concerted effort on the part of rare disease advocacy groups, patients and their families across the country to equalize access.

“We are excited to see the momentum building among our government partners across the country to increase access to treatments that improve the day to day lives of thousands of Canadians,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We will continue working alongside our clients to increase the understanding of our community’s needs on the national stage, ensuring that all Canadians, regardless of geography, have equal access to treatment.”

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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 www.muscle.ca or call our toll-free number at 1-800-567-2873.

MEDIA CONTACT INFORMATION:

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

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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

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Muscular Dystrophy Canada applauds Quebec for approval of SPINRAZA

For Immediate release – December 19, 2018
Toronto, Ontario

Muscular Dystrophy Canada applauds Quebec for approval of SPINRAZA for patients impacted by Spinal Muscular Atrophy Types 1, 2 and 3

Recommendations provided by the Institut national d’excellence en santé et en services sociaux (INESSS), the regulating body in Quebec, have resulted in the approval of SPINRAZA (nusinersen) for Spinal Muscular Atrophy (SMA) Types 1, 2 and 3 patients in that province.

This decision is the result of a refiling of the drug approval by Biogen early in 2018, after being reviewed and approved for use in Type 1 patients only.

The effectiveness of SPINRAZA was established in the pivotal ENDEAR and CHERISH studies and supported by multiple clinical studies. For individuals with infantile-onset (Type 1) and presymptomatic SMA results showed marked improvements in muscle function. How spinraza works

“Muscular Dystrophy Canada (MDC) was pleased to support both the initial and subsequent reviews by providing a comprehensive patient submission. Ensuring our community has a strong and credible voice in all matters related to trials and treatments is at the forefront of our work in Canada,” commented Muscular Dystrophy Canada CEO, Barbara Stead-Coyle. “This is a life changing treatment for our SMA community. We applaud the Minister of Health and Social Services Danielle McCann, and the Quebec government for reconsidering their initial decision and expanding access for more clients in Quebec.”

Stead-Coyle added “MDC’s commitment to ensuring SMA clients can access this treatment at an affordable price remains steadfast. There is still much work to do and we will continue to work tirelessly to ensure that all patients who may benefit from Spinraza will receive access as quickly as possible in Canada.”

A review from the Canadian Agency for Drugs and Technologies in Health (CADTH), the regulating body for the rest of Canada, is expected in the new year.

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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 visit www.muscle.ca or call our toll-free number at 1-800-567-2873.

MEDIA CONTACT INFORMATION:

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

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The Parent-of-Origin Phenomena in Congenital Muscular Dystrophy

CMD-resizeMuscular Dystrophy Canada is passionate about finding treatments and improving the quality of life for Canadians affected by over 150 different types of neuromuscular disorders. In order to do this, we have been dedicated to funding biomedical and discovery research to increase our understanding about these genetic disorders. Research has been successful at uncovering the causes and mechanisms behind several neuromuscular disorders, like Duchenne muscular dystrophy, there are still many unanswered questions with other conditions, like myotonic dystrophy.

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New therapeutic development showing promise for infants with SMA

gettyimages-86802223The early cancellation of the clinical trial for the experimental drug nusinersen found that the difference in the children given the drug, and those on the placebo was so drastic that doctors determined they could no longer administer the placebo in good conscience. The drug nusinersen is designed to help those affected by spinal muscular atrophy (SMA).

Dr. Craig Campbell is a scientist/paediatrician with the Children’s Hospital at London Health Sciences Centre who works closely with patients affected by SMA, and has seen first hand the encouraging signs the treatment has given some of his patients. We asked Dr. Campbell a few questions regarding the news of this clinical trial:

What is nusinersen and how does it work?

Nusinersen is an antisense oligonucleotide, which is basically a small piece of genetic material that works by tricking the cell machinery into reading and producing a normal SMN protein, from another gene that is very similar to the one missing in patients with SMA.

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