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Archive: April, 2019

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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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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.

With this decision, Saskatchewan has become the second province in Canada, following Quebec, to grant broad access to SPINRAZA™ for SMA patients. In March 2019, the Canadian Agency for Drugs and Technologies in Health (CADTH) provided a revised and expanded recommendation for access to SPINRAZA™ for SMA patients which include pre-symptomatic, Type I and Type II (with exceptions and limitations). Notably all patients over 12 years of age and all those who have reached the ability to walk independently (Type III) are denied treatment according to this recommendation. The provincial jurisdictions are ultimately responsible to define their coverage criteria and Biogen Canada is working with all these jurisdictions to provide broad access to SPINRAZA™ to patients that need it. The decision of the Government of Saskatchewan is further demonstration after 45 other countries and the province of Quebec that an ongoing and sustainable solution for covering broadly this life-long treatment is possible and can be implemented in all Canadian jurisdictions.

Biogen Canada is working diligently to find solutions and will continue to pursue and advocate for broad and sustainable funding for all SMA patients in Canada. Patients living with SMA deserve equal opportunity to receive access to SPINRAZA™ and Biogen remains committed to working with each provincial government outside of Quebec and Saskatchewan until broad access is granted to patients.

Sincerely,
Biogen Canada

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Read the news release

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.”

Both projects focus on improving a critical component, the delivery vehicle, used in gene therapies like gene transfer and gene editing.

“Jesse’s Journey is proud to partner with Muscular Dystrophy Canada to fund these two exceptional research studies,” said Perry Esler, Executive Director Jesse’s Journey. “For nearly 25 years, Jesse’s Journey has funded the most promising Duchenne research worldwide and we’ve seen first-hand the difference that is made in the life of a person facing Duchenne when they are able to benefit from scientific discovery. Jesse Davidson – the inspiration behind Jesse’s Journey – once said, “When researchers work together, miracles can happen.” We believe the same can be true when funding organizations work together, and it’s in the spirit of collaboration we welcome the opportunity to join forces with MDC.”

There are already several clinical trials exploring gene transfer (a type of gene therapy) underway for Limb-Girdle and Duchenne Muscular Dystrophies. These make use of AAV (adeno-associated virus) as the transport vehicle but limitations exist in using AAVs including small packaging capacity, difficulty in producing large amounts, and potential to cause an immune response especially if they need to be administered more than once.

To circumvent some of the limitations of AAV, both research projects will be testing alternative non-viral vehicles.

Dr. Gramolini and his team will test BIPHIP, a nanoparticle which will be used to transport CRISPR gene editing tools into cells. with the goal of editing a genetic change that causing this disorder. Dr. Rudnicki and his team will test exosomes as vehicles to deliver the Wnt7a gene, which has been shown to help muscle cells regrow.

If successful, the results could have broader applicability to other types of muscular dystrophies.

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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.

ABOUT JESSE’S JOURNEY

Jesse’s Journey is a partnership of science and families dedicated to the discovery of treatment and cure of Duchenne muscular dystrophy – because parents should not out live their children. To learn more about Jesse’s Journey visit www.jessesjourney.com or call 519-645-8855.

MEDIA CONTACT INFORMATION:

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

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