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Ontario Leadership Conference Highlights

on-confOn Saturday, November 5, 2016, Muscular Dystrophy Canada hosted its’ Bi-Annual Ontario Volunteer Leadership Conference at the Holiday Inn Toronto International Airport. This one-day educational and informative conference featured various speakers and presentations that were both motivating and inspiring.

Buzz Green, Chair of the Board, welcomed everyone and introduced CEO, Barbara Stead-Coyle, who presented an organizational overview and talked about the strategic plan for the next three years. Services staff highlighted how donor dollars are used to provide services to those affected by neuromuscular disorders and how we help to enhance the lives of those affected.

on-conf-possible-2Those who attended were very excited to hear research updates from Dr. Robin Parks, Dr. Jodi Warman, Dr. Rashmi Kothary and Dr. Ronald Cohn and enjoyed learning about some of the recent research advancements. Throughout the conference, personal and emotional stories were shared by various volunteers, clients, Fire Fighters and partners. A highlight of the afternoon was when Luca “Lazylegz” Patuelli asked 11 year old Kaleb to join him for a break dancing lesson.

The banquet opened with special guests being piped in by the Toronto Fire Pipes and Drums Band. Genuine and heartfelt speeches during the banquet were given by Alex Harold, Christina Massad and National Ambassador Danielle Campo-McLeod. Several of our dedicated volunteers and partners were also recognized with annual awards.

Thank you to all who attended and for making the day so memorable, and congratulations to our award winners.

Ontario Chapter of the Year Award
Ottawa Chapter

Ontario Dr. George Karpati Award
University of Ottawa Centre for Neuromuscular Disease

Ontario Fire Department of the Year
Sault Ste. Marie Professional Fire Fighters Association

Ontario Fire Fighter of the Year
Launie Fletcher

Danielle Campo Extra Mile Award was presented to:
Middlesex Centre Fire Department – Coldstream Station
Russell Fire Department
The Espanola Fire Fighters
Whitby Fire and Emergency Services
Ottawa Fire Services – District #7 Fire Fighters

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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.

 

What improvements were seen in the clinical trial?

In both the infant and child study (SMA type 1 and 2 respectively) the motor skills were better than those treated with placebo. At this point we have not seen all the specific numbers, but for example in the SMA type 2 study the children actually improved over their baseline function by about 4 points on a motor scale we use to measure function in children with SMA.  The placebo group declined by 2 points during the same 15 month time frame. It is very unusual for children with SMA to improve on this score as SMA is typically a disorder where patients decline in function over time.

 

How will the development of nusinersen continue now that the clinical trial showed a positive result?

All children on the trials have been moved over to open label extension studies, and the company has opened up a special access program so that all children with type 1 SMA  can get access to nusinersen free of charge.  Currently the investigative sites in Canada (Vancouver, London, Toronto, and Montréal) are taking referrals for SMA patients to get nusinersen.  We anticipate that the company Biogen Inc. will be applying soon to Health Canada for a Notice of Compliance for a new drug.

 

Could a clinical trial for nusinersen open for adults affected by SMA?

I think it would be unlikely to happen any time soon. The company will probably apply for a wide label for the drug, and thus a trial for adults may not be needed. In the case that Health Canada limits the label to a specific age group, then a trial in adults may be needed.

 

What does this mean for research into other neuromuscular disorder treatments?

This has definitely helped give some enthusiasm and hope for the whole neuromuscular community, and it specifically gives some validity to the use of AON, which have been under trial in Duchenne MD (Eteplirsen and Drisapersen) and in Myotonic dystrophy (Ionis).  However, it raises the question about how other medications can be tested in children with SMA now that an effective drug is on the market.  Will placebo trials be possible, will there be enough children willing or able to participate in new trials, how will regulatory agencies respond to new trial applications? These are all not clear right now.

 

What are your hopes for the future use of this drug in patients with SMA?

We hope that we get Health Canada approval for a new drug in an accelerated manner, and that a thoughtful label is applied.  Also we hope that a clear plan for coverage of the drug is established so that families do not have to pay or worry about access due to financial resources. Ideally this will be full government coverage, but other options will be important to explore such as managed access schemes.

 

Watch the CTV News feature video below, and read the accompanying article here.

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How I See It: Hello 2017

hello-20172016 was a year full of lessons for me. I spent most of the year trying to figure out who I was, where I wanted to go, and questioning what I was doing with this life. It was the first full year I spent away from my family. There were many nights spent staying awake, questioning every decision that lead me here.

It was a very difficult year – probably the most difficult I have ever experienced. It was both mentally and physically draining. It got to a point where I wasn’t sure what I was going to do, and how I was going to go on. Months would pass by where I didn’t write a single word, and I just didn’t have the energy or motivation to do it. I stopped reading; I let my hobbies fade away. I complained about everything, and I found myself showing hatred more than kindness. Sometimes we hit a brick wall, and I think 2016 was the year I finally hit mine.

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INTERNATIONAL DAY OF PERSONS WITH DISABILITIES: Lana on Goal #9

unsustainabledevelopmentgoals_brand-01The third of December marks the United Nations International Day of Persons with Disabilities. This year’s theme is the 17 Sustainable Development Goals, which were adopted by countries all over the world on September 25th, 2015. These goals look to move towards the ending of  poverty, protecting the planet, and ensuring prosperity for all as part of a new sustainable development agenda.

We asked our friend and colleague Lana Miley to speak to one of the Sustainable Development Goals and tell us what it means to her as someone who lives with a disability. Lana chose goal number nine: Industry, innovation and infrastructure. Here’s what she had to say about it:

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CDA Consultations: National Youth Forum

peelertrudeau Alex Peeler wrote for us about his petition for a National Assistance Program for Persons with Disabilities. This was shortly before he was heading to Ottawa to participate in the National Youth Forum for the CDA consultation process. Here he recounts his experience:

I applied for the National Youth Forum in August after hearing about the consultations for the Canadians with Disabilities Act (CDA) through Muscular Dystrophy Canada. I decided to apply because I have always been an active advocate for persons with disabilities and saw this as an opportunity to put my passion for advocacy to good use. Then, in early October, I received an email from the Minister of Sport and Disability, Carla Qualtrough, saying that I had been chosen to participate in the consultations. I was very excited and proceeded to make the necessary arrangements for my trip to Ottawa.

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