Dear Canada’s SMA community,

Navigating life with SMA can often be challenging – but also rewarding. As a community, we are always looking to lift one another up and share real stories to ensure families know they are not alone.

That is why we are excited to share a recent launch of a new website and support hub for the SMA community – SMA My Way Canada

SMA My Way Canada is part of a global community. The website offers a platform to learn from and engage with people sharing real and honest experiences, stories and perspectives of those living with SMA, as well as their families, friends, and caregivers.

Stories shared by the website’s contributors cover a number of areas important to our community including accessibility, adulting, relationships, hobbies, parenting and everyday life. You can learn more on the website and from its contributors about:

● Tips and tricks for navigating international travel

● Planning for accessibility changes and challenges in the home as your child grows older

● Breaking down stereotypes surrounding sexual intimacy and relationships for those living with a disability

● The importance of finding ‘me-time’ as a caregiver

And much more!

Cure SMA Canada welcomes this website as a community support tool for all those affected by SMA in Canada. We encourage you to visit SMA My Way Canada to learn more and engage with contributor stories and experiences. We certainly will be!

Sincerely,

Susi Vander Wyk

Executive Director

Cure SMA Canada

Dear SMA community,

We are excited to share the recent launch of a new website for the SMA community – SMA My Way Canada.

 

SMA My Way Canada is part of a global community. The website offers a platform to learn from and engage with people sharing real and honest experiences, stories and perspectives of those living with SMA, as well as their families, friends, and caregivers.

 

The stories featured on the site shared by its contributors covers a variety of areas important to our community including accessibility, adulting, relationships, hobbies, parenting and everyday life. You can learn more from the website’s contributors including tips and tricks for international travel with a power wheelchair, how to create safe spaces for those with disabilities to speak about intimacy and relationships, the importance of finding ‘me-time’ as a caregiver and much more.

 

Cure SMA Canada welcomes this website as a community support tool for all those affected by SMA. Platforms like SMA My Way will help Canada’s SMA community to strengthen its collective voice, one story at a time.

 

Sincerely,

Susi Vander Wyk

Executive Director

Cure SMA Canada

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‘About SMA – Resources’

Useful Links

SMAMyWay.ca – support hub for Canada’s SMA community where contributors share their stories and experiences across a variety of topics including accessibility, adulting, relationships, hobbies, parenting and everyday life.

This video was created with the intention of educating SMA families, and the general public, about the important role bulbar function testing plays in the treatment journey of a child living with SMA. With the help of the Sousa family we were able to convey Chloe’s journey in a fun and innovative way while tackling our goal.

Treatment potential for our pediatric and adult patients!

Dear Canada’s SMA community,

Cure SMA Canada is happy to share that approval has been issued by Health Canada for Risdiplam for SMA patients 2 months of age and older.

Risdiplam is an oral treatment from Roche that treats SMA by increasing the SMN levels. The convenience and benefits of this oral treatment has proven to be life changing for Canadian patients that are already accessing through clinical trials or through Canada’s Expanded Access Program.

Cure SMA Canada looks forward to working with stakeholders to provide justification and support for the various government levels of approval. Our ultimate goal is to ensure that Canada’s SMA patients will have the opportunity to access Risdiplam in a timely manner. Many of Canada’s SMA patients are still living without access to treatment, Risdiplam offers a solution to the unmet need.

We look forward to engaging the SMA community to support our initiatives as we continue our advocacy for access to treatment.

Health Canada’s approval is a wonderful first step on our way to access Risdiplam, we look forward to ensuring Canada’s SMA patients will benefit from this life changing treatment.

Sincerely,
Susi Vander Wyk
Executive Director
Cure SMA Canada

March 30, 2021

 

As requested, and as part of our ongoing commitment to keep you informed of important milestones regarding Zolgensma® (onasemnogene abeparvovec), I am writing on behalf of Novartis to let you know that on Friday March 26^th, the Canadian Agency for Drugs and Technologies in Health (CADTH) published their recommendation. Zolgensma has been recommended for reimbursement by public drug plans outside of Quebec for the treatment of pediatric patients who meet the following criteria:

 

• Genetic documentation of 5q SMA with biallelic mutations in the SMN1

 

• Symptomatic or pre-symptomatic with 1 to 3 copies of the SMN2

 

• Aged 180 days (6-months-old) or younger.

 

• Do not currently require permanent feeding or ventilatory support (either invasive or non-invasive).

 

While we welcome a positive recommendation, Novartis is disappointed that it is limited by age, without a mechanism for case-by-case review. The societal impact of unequal access due to delayed diagnoses for all SMA patients across Canada should have been a key consideration as we saw in the recommendation from the Institut national d’excellence en santé et services sociaux (INESSS), Quebec’s health technology assessment body.

 

Following Health Canada approval and the closure of the global managed access program to new enrollments in Canada, interim access for Zolgensma was put in place so that families affected by SMA would not be caught in limbo while we work through the formal reimbursement process. It’s troubling that families must continue to fight for treatment when all jurisdictions have the ability to provide Zolgensma now.

 

We anticipate the CADTH recommendation will be frustrating for the community, particularly as newborn screening for SMA has yet to be widely adopted across the country. Know that we will continue to advocate for the broadest possible access to Zolgensma, as we negotiate a final agreement for all Canadians with the pan-Canadian Pharmaceutical Alliance.

 

Sincerely,

 

Brent Warner

 

Vice President, Gene Therapy and Rare Diseases

 

Novartis Pharmaceuticals Canada Inc.

 First baby in Canada identified with SMA using newborn screening: given early treatment, ‘loves to move’

Dec. 2, 2020—OTTAWA— When Newborn Screening Ontario diagnosed spinal muscular atrophy (SMA) in Aidan Deschamps’ bloodspot, he became the first baby in Canada identified with the genetic, neuromuscular condition through a provincial newborn screening program.

 

Just six days before his birth, Newborn Screening Ontario (based at CHEO) started testing for SMA using the heel-prick test obtained from babies born anywhere in the province. This means infants are diagnosed within days of birth, so treatment can start as soon as possible.

 

During a virtual news conference today, the family gave us a peek into how Aidan is doing and what the early identification of his SMA has meant for them.

 

Their journey started when Aidan was 10 days old and his parents, Amanda Sully and Adam Deschamps, received a call from Newborn Screening Ontario. Aside from just getting over jaundice, which is fairly common in newborns, Aidan was doing well. So, the call was a surprise.

 

“They told us Aidan had screened positive for spinal muscular atrophy and had scheduled an appointment for us with Dr. Hugh McMillan, a neurologist at CHEO,” said Amanda. “We had no idea what SMA was and what it might mean for our family.”

 

SMA is a debilitating and potentially deadly neuromuscular disease. While officially considered a rare disease, affecting 1 in 10,000, it is the most common cause of inherited death in childhood. Children with the most severe infantile form rarely live beyond their second birthday if left untreated. Symptoms arise in these most severe cases before six months of age.

 

Until recently, there were no therapies for SMA. But now there are, which paved the way for the genetic condition to be added to Ontario’s newborn screening program.

 

“When I started at CHEO in 2010, we had nothing that could treat SMA,” said Dr. Hugh McMillan, a pediatric neurologist at CHEO, clinical investigator at the CHEO Research Institute and associate professor at the University of Ottawa. “We managed symptoms as best we could to give children their best lives possible. Now, we actually have options for treatment and more therapies are on the near horizon. It’s very exciting. Being able to significantly improve the lives of kids like Aidan is unbelievably rewarding, as a physician and a researcher.”

 

Significant and early research on SMA was done by CHEO researchers, led by Dr. Alex MacKenzie, who discovered a gene called NAIP, which may contribute to the severity of the disease. In addition, Dr. McMillan was a clinical investigator for one of the therapies now available.

 

One therapy is called Spinraza, which is a therapy that corrects how the faulty gene produces certain proteins. It is approved in Canada and available in Ontario. It requires ongoing treatments throughout the person’s life. The second is a gene replacement therapy called Zolgensma, which is approved in the U.S. but not Canada and has been made available through the pharmaceutical company’s managed access program. It only needs to be given once.

 

“The neurological damage caused by the most severe forms of SMA starts very early in life and once the damage has occurred, it can’t be reversed. So the sooner a child with SMA accesses therapies, the better they do. This is why newborn screening is so important,” said Dr. Pranesh Chakraborty, Chief Medical Officer of Newborn Screening Ontario. “It helps us find babies with SMA as early as possible.”

 

Aidan was quickly started on Spinraza at the age of three weeks before being granted access to and given Zolgensma when he was five weeks old.

 

“Because Aidan was treated before he had any symptoms of the disease, we can’t say for certain what the severity of his disease would have been. The very real hope is that Aidan has been treated early enough to maximize his developmental potential,” added Dr. McMillan.

 

And what does this all mean, today, for Aidan and his family?

 

“We are overjoyed to report that Aidan is meeting all of his milestones,” said Amanda. “He’s such a happy little person and he truly loves to move! We are aware that nothing is certain about Aidan’s future but we are beyond grateful to everyone who helped give Aidan the greatest chance possible to live his best life. He means the world to us.”

The COVID-19 pandemic, as you may well know, is taking a particularly heavy toll on Canadians with disabilities.

So now, more than ever getting approved for the Disability Tax Credit or CPP Disability makes a huge difference to the lives of disabled Canadians and their families.

We think your team and those you are helping can benefit greatly from the guides and resources on our website so I wanted to bring it to your attention, hoping you can share them with your community:

Disability Tax Credit Guide

CPP Disability Guide

Please feel free to contact me with any questions, or if you are looking for more information.

P.S. We are offering a $1,000 scholarship to young Canadians with disabilities who are pursuing post-secondary education at an accredited Canadian institution.

Read about last year’s recipient, Katarina V., a young woman from Surrey, BC living with ASD.

Regards,

Jay Henson

Families of Alberta children suffering from spinal muscular atrophy (SMA) may now be eligible to receive funding for gene replacement therapy treatment.

Media inquiries

Steve Buick

780-288-1735
Press Secretary, Health

Cure SMA Canada is inviting you to a scheduled Zoom meeting.

Topic: Cure SMA Canada – Treatment info and updates

Time: May 7, 2020 09:00 Pacific Time (US and Canada)

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