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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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$4.8 Million and counting….

Shad’s 44th annual golf tournament raises $175,000 for Muscular dystrophy

Since 1973, Shad’s R&R Golf Tournament has supported Muscular Dystrophy Canada’s efforts to improving the quality of life for the tens of thousands of Canadians with neuromuscular disorders and taking the lead in research for the discovery of therapies and cures.

This year, more than  200 participants from all sectors of the automotive aftermarket took part in a day of golf, dinner, and prizes with all proceeds going to Muscular Dystrophy Canada. The 44th annual Shad’s R&R for muscular dystrophy raised $175,000, bringing the total money raised since its inception to $4.8 million.

The proceeds from this golf tournament have been invested in world-class research projects that are helping to answer questions about neuromuscular disease and finding ways to improve the medical care and quality of life for the young children that are affected by these conditions. For the first time in history we are seeing new treatments and clinical trials that show incredible potential to slow or reverse the progression of some of the 160 different types of neuromuscular disorders. Now more than ever, people with neuromuscular disorders are living longer and more empowered lives.

All of this would not be possible without the ground breaking research taking place throughout Canada and beyond. This research would not be possible without donors and supports, like Shads, who continue to demonstrate incredible generosity.A very heartfelt thank you to the organizing committee including David, Keith Brad, Marilyn, Samantha and everyone involved. Your dedication to this cause is evident and your dedication so greatly appreciated.

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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Reflections on Rare Disease Day 2017

rdd-logo-300x287Today, February 28, 2017 is International Rare Disease Day (RDD). While it is just one day on the calendar, people affected by rare diseases are impacted by their condition 24 hours, 7 days a week, 365 days a year.

A rare disease is defined as a medical condition that impacts a small percentage of the population, less than 1 in 2000 people.

• 80% of rare diseases have identified genetic origins whilst others are the result of infections (bacterial or viral), allergies and environmental causes, or are degenerative and proliferative.
• 50% of rare diseases affect children.

Muscular Dystrophy Canada supports research to find treatments for over 150 different types of rare neuromuscular disorders. You see, muscular dystrophy isn’t just one disorder; Different genetic mutations cause different disorders. While investments in research over the past decade have resulted in a tremendous increase in our knowledge, and understanding of the molecular causes of neuromuscular disorders, we still have a long way to go.

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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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