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The Power of Giving Back

GivingTuesday – the global day of giving

Join us for the 7th annual GivingTuesday on December 3, 2019!

GivingTuesday is a global day of giving that happens each year after Black Friday and Cyber Monday. It’s a time when Canadians, charities and businesses come together to celebrate giving and participate in activities that support charities and non-profits. There is no “right” way to participate as long as it supports generosity and giving. Donate, volunteer time, help a neighbour, or spread the word.

GivingTuesday was started in Canada by a group of organizations including GIV3CanadaHelps.org, and now includes over 6,500 partners. GivingTuesday was originally started in the US in 2012 by the NYC 92Y and several other community organizations.

Make a donation to La Force DMD here –  or visit our facebook event page here

 

What was achieved in 2018?

Last year, an estimated six million Canadians took part in GivingTuesday and found ways to ‘Do Good Stuff’. Today, as the holiday season is fast approaching,

Now in its sixth year, GivingTuesday kicks off the beginning of the charitable season with more than 6,500 partner organizations registered in Canada and over 40 community movements active in cities and towns across the country.

 

Countdown

 

GivingTuesday Countdown

Help someone afflicted Duchenne muscular dystrophy (DMD)

What is Duchenne? It’s a degenerative muscle disease, afflicting mostly young boys 1-3500. It’s a fatal disease with no cure, taking muscles strength away and leaving young adults in a wheelchair and premature death in their late twenties. Many treatments are on the horizon, but we need your help to raise awareness and funds because there is no time to lose for people afflicted with DMD. Our organization raises funds for promising research and raises awareness to have a unified DMD community across Canada to access new treatment quickly.

 

How to contribute to our cause:

There is no small amount!

Our organization aims to unite the DMD community to raise awareness around a common objective: that of providing access to new treatments as fast as possible and to participate in the funding of promising research projects. We also raise funds for two promising research-based in Canada.

Make a donation to La Force DMD here –  or visit our facebook event page here

 

Other ways to support us:

  • Organize a fundraiser or a sporting event
  • Buy our promotional items to make gifts
  • Share our stories, video and article
  • Display our colours during your outings and events.

 

More links

A DMD gene therapy has been placed on clinical hold

Following the occurrence of a safety incident, the FDA has placed on hold the clinical trial for SGT-001, the Solid’s gene therapy candidate for Duchenne muscular dystrophy (DMD). This clinical hold is the second bad news the DMD community has received this month. Last week, Swiss pharma giant Roche announced it was terminating its study of an investigational anti-myostatin adnectin protein in ambulatory boys with DMD. Roche said an analysis of the ongoing data indicated that its treatment RG6206 was “highly unlikely” to demonstrate clinical benefit in the trial.

 

La Force is sharing this press release provided by Solid Biosciences, Nov. 12, 2019,> PRESS RELEASE <

 

Solid Biosciences Provides SGT-001 Program Update

Solid Biosciences Inc. provided a clinical update on SGT-001, a microdystrophin gene transfer therapy, and reported that the U.S. FDA had notified the company that IGNITE DMD, its Phase I/II study of SGT-001, has been placed on clinical hold. 

To date, six patients have been dosed with SGT-001, Solid’s gene transfer candidate under investigation for Duchenne muscular dystrophy (DMD). This includes three patients in the first cohort, who continue to do well and are being followed per the study protocol. Three patients were subsequently dosed in the second cohort. The first two of these patients are also doing well and being followed per study protocol.

The third patient in another cohort, dosed in late October, experienced a serious adverse event (SAE) deemed related to the study drug that was characterized by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. Neither cytokine- nor coagulopathy-related abnormalities were observed. Currently, the patient is closely followed by his care team. He is recovering and continues to improve.

The company reported the event to the FDA and the study Data Safety Monitoring Board (DSMB). The FDA has notified the company that the study has been placed on clinical hold. Solid will work with the FDA in an effort to resolve the hold and determine the next steps for IGNITE DMD. The company continues to plan to report additional biomarker data from the study before the year-end.

 

Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences – “We are encouraged that this patient is recovering. I would like to thank both the patient and his family for their participation in our study, as well as the team at the University of Florida for the excellent care they provide. We remain committed to bringing meaningful new therapies to the Duchenne community and continue to believe in the differentiated construct of SGT-001 and the potential benefits it may offer to patients. In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001. We look forward to sharing this additional data and working with the FDA to resolve the clinical hold and determining next steps for the program.”

 

Last year, the FDA placed a clinical hold on the trial following the report of a serious adverse event. Solid Biosciences Announces Clinical Hold On SGT-001 microdystrophin gene transfer Clinical Phase I/II Clinical Trial for Duchenne Muscular Dystrophy.  That hold was lifted in June 2018 after the company addressed the FDA’s concerns.

About SGT-001

Solid’s lead candidate, SGT-001, is a novel adeno-associated viral (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD, mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of DMD, regardless of genetic mutation or disease stage.

SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, in the United States and Orphan Drug Designations in both the United States and European Union.

Learn more here

In case you don’t remember the specifics about the microdystrophin and gene therapy we invite you to watch the interview we conducted in London with Dr. Jeffrey Chamberlain PH.D.:  Here

About Solid Biosciences

Solid Biosciences is a life science company focused solely on finding meaningful therapies for Duchenne muscular dystrophy (DMD). Founded by those touched by the disease, Solid is a center of excellence for DMD, bringing together experts in science, technology and care to drive forward a portfolio of candidates that have life-changing potential. Currently, Solid is progressing programs across four scientific platforms: Corrective Therapies, Disease-Modifying Therapies, Disease Understanding and Assistive Devices. For more information, please visit www.solidbio.com.

 

More links

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Teaming up to take down Duchenne

Research and collaboration are bringing Canadians closer to a cure than ever before. However, many parents fear that access to treatment may come too late for their child.

by Denise Deveau, Sep 6, 2019, > published on Maclean’s

 

 


For Nicola Worsfold and her husband Ed, the day they were told their six-year-old son Owen had Duchenne Muscular Dystrophy (DMD) is one they’ll never forget.

 

Nicola Worsfold – “It was like someone took a huge sledgehammer and knocked us off our feet. We found out that there was nothing we could do because, at the time, there was no cure or medication that could help.”

 

A progressive muscular disorder, DMD is caused by a genetic mutation that prevents the body from producing the structural protein needed to protect muscle cells. The Canadian Neuromuscular Disease Registry estimates that there are less than 800 cases in Canada, with almost all affecting boys.

As it stands today, DMD is fatal. A child with the disorder becomes weaker over time until they succumb to the disease in their early to mid-twenties. It can be inherited or, as with Owen’s diagnosis, the result of a random spontaneous mutation.

 

Read the full article here

Living with DMD, Carl’s resilience

Notes from the “Living with DMD” web-doc Tecima Productions team. Carl’s resilience.

Our small production team travelled across Canada, meeting with families whose children have Duchenne muscular dystrophy (DMD). We met with Dakota (age 6), Anakin (age 11) and Carl (age 35) at critical moments in their journey. Our goal was to produce a Web-doc in three acts, to raise awareness about DMD by living it through the eyes of the families suffering because of it. This degenerative muscle disease, for which there is no cure, usually leads to premature death in the patient’s early twenties … But, guess what? … this isn’t always the case, because Carl Marier has defied all odds and is now aged 35. When we met him we were overwhelmed by his joyfulness, his presence, and his sense of humour, even if he is the youngest person living in a residence for the elderly. Living with DMD: Carl’s resilience story.

 

Surviving 3 major operations

Today, Carl is connected to his respirator 24/7; the respirator sends the air directly to the lungs, a tracheotomy allows him to limit the colds that could turn into pneumonia and his gastrostomy helps him to eat. As he grew older, it was harder and harder for his family to care for him until it became impossible as he needed to cared for day and night. Because of a lack of adapted facilities, their only option was to send him into a residence for the elderly.

 

Alain Marier, from the documentary  Living with DMD– ” We had to place Carl, who is 34 years old, in a residence for seniors. I’ll tell you, almost three-quarters of the seniors there do not know where they are. They have Alzheimer’s or dementia. He’s the baby in the place because he’s the youngest.”

 

The power of a positive attitude

When we asked him what quality he developed because of DMD, Carl immediately answered “resilience”. He greeted us in his room with a great amount of energy and punctuated our visit with several jokes. He also told us he has many friends and he loves to socialize with them in the residence. We also witnessed a tender relationship between him and his step-mother, Sonia Gélineau.

 

Sonia Gélineau – from the documentary Living with DMD– “Carl came into my life when he was nine-and-a-half years old. Later, in 2001, his father and I were married. Living with the disease is not always easy”.

 

Faced with a great deal of suffering and inevitable destiny, what we witnessed from our encounter with Carl was how the strength of character, a solid positive attitude, and unfailing love link together in the face of adversity that is difficult to imagine.

 

Enjoy the web-doc. Thank you for sharing.

Dakota’s big smile – A race against time to access new treatments

The documentary “Living with DMD” is produced by www.tecima.com

 

A Race Against Time to Access New Treatments

Duchenne muscular dystrophy seen through the eyes of the families

There are over 7,000 rare diseases in Canada. Duchenne muscular dystrophy (DMD) is one of them. DMD afflicts children, especially young boys, and sometimes girls. This degenerative muscle disease causes loss of the ability to walk, heart and respiratory failure, and usually leads to premature death in the early twenties. Tecima Productions produced the “Living with DMD” documentary to tell the story of people living with DMD and their families at crucial moments in their journey: those of Dakota, age 6, Anakin, age 11, and Carl, age 35.

Anakin’s Loss of Autonomy

For other children, being 10 years old is a period for developing and gaining independence. For 10-year-old (in 2018) Anakin who has DMD, it’s a different experience, as he is progressively losing autonomy and is forced to seek help from his parents to get out of bed, in and out of the car, etc. It’s also during this exact stage that he becomes fully conscious of his destiny.

 

Bruce Babington, an excerpt from the documentary Living with DMD “The last year has been tough for him. The disease has progressed quite significantly, and he’s losing the ability to use his legs. He cannot walk great distances, and he’s struggling to maintain the strength he has in his legs. You can see that it’s beginning to affect him both physically and mentally.”

 

A Race Against Time to Access New Treatments

Rare diseases such as DMD are fraught with all sorts of obstacles for families. There is very little funding for research, it is difficult to access new treatments, and long-term care for young adults is costly and often not available to families.

 

Marie-Catherine Du Berger excerpt from the documentary Living with DMD “It’s a bit of a race against time because the faster you get the treatment, the better it is. But there are many obstacles in the drug approval process here, with repayments, financing, etc. By the way, it is done for us.”

 

Read the story about Bruce Babington, who cycled across Canada in July 2018 to raise funds and awareness for DMD.

 

Help Us Raise Awareness!

After reading this article or watching our documentary, you can certainly feel the urgency of sharing the story of these three families. We want to educate the public about the realities of people living with DMD, on behalf of those who have suffered, those who suffer now, and for future generations. Our aim for this project is to get better funding for research, to make new treatments more easily accessible, and so that long-term care becomes better available to young adults. We are doing this so that they have a chance for a better life.

How can you help us? Watch our documentary, continue reading our article, and share them with your communities. Every action counts.

Enjoy viewing our documentary and thank you for sharing it in your community

 

The “Living with DMD” documentary was produced by www.tecima.com