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
Eight years ago, I sat in a doctor’s office staring down at a brochure from Muscular Dystrophy Canada mindlessly reading the same line over and over again.
Alfred and his son, Eloi who was diagnosed with Duchenne Muscular Dystrophy need your support. Alfred’s passion for helping others led to him being named an honorary firefighter.
I was in shock. A doctor had just told me my 4-year-old son, Eloi, had Duchenne Muscular Dystrophy. When news like that comes, it’s like the world stops and the sky is falling on you.
All I could think about was that my son was going to stop walking and I would live to bury him and there was little I could do to intervene.
I didn’t know in that moment I would have supporters like you on my side, to help my family through this.
But then we reached out to Muscular Dystrophy Canada (MDC) and learned about the incredible support they are able to provide thanks to donors. In fact, we were so moved that we decided to take action by helping them raise much-needed funds for equipment, research and support for other famliles impacted.
September is Muscular Dystrophy Awareness Month so I hope that you, too, will take action by making a gift today to help support the more than 50,000 Canadians impacted by neuromuscular disorders.
Families like Alfred’s, impacted by muscular dystrophy, need your support.
Because donors like you are having an incredible impact on the lives of individuals and families, like mine, who are living with a muscular dystrophy diagnosis.
For example, thanks to donors, we were able to adapt our home so Eloi can safely and freely get around. He has a scooter. He’s gone to summer camp. And, MDC was even able to visit his elementary school with their Muscle Facts program to help his classmates and teachers better understand his diagnosis and challenges. None of this would have been possible, without your support.
Sadly our story is not unique. Every year, more families have to figure out what a diagnosis of muscular dystrophy means for their son, daughter, brother, mother. You may even be one of those people, and you remember that moment as vividly as I do.
Mostly, I remember feeling helpless. The fear of the unknown can be so overwhelming. But joining the Muscular Dystrophy Canada family has been one of the most transformative experiences of my life. Not only has my family been gifted with new friendships and an invaluable support network – I’ve been able to help other families.
Donors, like you and me, can improve the lives of the thousands of Canadians living with a muscular dystrophy. That’s why I hope you will mark Muscular Dystrophy Awareness Month with a generous gift.
I’ve seen first-hand how gifts from donors play a critical role in supporting individuals. And because of my job, managing clinical trials for a pharmaceutical company, I’ve seen how your gifts are moving promising research forward as well. Research can make the difference between an individual, like my son, walking or spending the rest of their lives in a wheelchair.
Right now, Eloi only needs a wheelchair for long distances. But we know the day may come when he will have to rely on it completely. And, he’ll require other supports to maintain his quality of life. This disorder is progressive. It can be slowed, but not stopped. That’s why ongoing donor support is critical.
By making a gift today, you will be providing hope to all Canadians, whether they have been living with a disorder or they are just getting a diagnosis. Please give generously.
From my family to yours,
Alfred Breton-Pare, Eloi’s Proud Papa
P.S. September is Muscular Dystrophy Awareness Month. Please make a gift to MDC so together we can fund life-changing equipment, and invest in life-saving research.
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.
Muscular Dystrophy Canada does not use telemarketing services.
Several years ago, the Canadian Fire Fighter Curling Association (CFFCA) retained a telemarketing company to assist in fundraising for their annual curling event. A portion of the funds raised in past years were donated to MDC.
Muscular Dystrophy Canada can confirm a campaign soliciting donations for the Canadian Fire Fighter Curling Association has begun, seeking donations that support the Canadian Fire Fighter Curling Association.
We have received complaints regarding the tone and approach of solicitation calls. We have brought these to the attention of the President of CFFCA.
Should you have any concerns or questions please contact the Canadian Fire Fighter Curling Association directly at cffca.ca. We sincerely thank all of our generous supporters for making our work possible.
NOTICE IS HEREBY GIVEN that the Annual General Meeting of the Members of Muscular Dystrophy Canada will be held at the:
Doubletree by Hilton
Toronto Airport Hotel
925 Dixon Road
Toronto ON, M9W 1J9
Receiving the following reports:
Chair of the Board and the CEO;
Treasurer; and
Independent Auditor, together with the audited financial statements for the year ended March 31, 2019.
Electing the Board of Directors
Appointing the Auditors
Other business as may properly be brought before the meeting.
Dave Ferguson
Secretary of the Board of Directors
Cowichan Bay, BC
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.
I met Brayden Graft when he was just five-days-old. Little did I know then the impact he would have on my life.
Individuals, like Brayden Graft, diagnosed with Duchenne Muscular Dystrophy need your support to live rich, full lives.
My friends Leanne and Tony had been fostering children for about five years and already had a baby at home when their social worker called about Brayden. They didn’t even think twice about taking him home.
You probably have friends just like Leanne and Tony. Humble, hardworking people with a ton of love to give. Leanne has always had a soft-spot for children, with four of her own, but as her kids grew up and left home she knew she and Tony had more love to give.
Eighteen-months-later they were offered the chance to adopt Brayden and once again they didn’t hesitate.
They couldn’t imagine life without him. He had become their precious, fun-loving son.
Just a couple of months later, Brayden was diagnosed with Duchenne Muscular Dystrophy (DMD).
This month, my team members and I celebrated International Fire Fighter Day, and in honour of that, I hope you will read Brayden’s story and consider making a donation to Muscular Dystrophy Canada (MDC).
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.
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.
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.
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
Dave Ferguson
