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Sarepta announces top-line results for its investigational gene therapy

Sarepta Therapeutics announces top-line results for part 1 of study 102 evaluating SRP-9001, its investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD)

 

Jan. 7, 2021, >Original press release<

Sarepta Therapeutics, Inc. announced top-line results from Part 1 of Study SRP-9001 (Study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) in 41 patients with Duchenne muscular dystrophy. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.

Quickly

  • The study met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, measured by western blot, in SRP-9001-treated participants versus placebo.
  • SRP-9001-treated participants showed an increase in NSAA total score compared to placebo at 48 weeks; however, the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post-treatment  
  • In the pre-specified analysis by age-group, by which the randomization was stratified, participants aged 4-5 years at the time of treatment with SRP-9001 demonstrated a statistically significant improvement in NSAA total score age-matched placebo cohort, achieving a 4.3-point improvement on NSAA at 48 weeks post-treatment from baseline.
  • No new safety signals were identified for SRP-9001, reinforcing the favourable safety profile observed to date.

Study 102 is ongoing and remains blinded to participants, investigators, site staff and sponsor staff with direct site interaction. All 41 participants have completed their Part 1, 48-week assessment and have entered the Part 2 crossover phase. Participants continue to be monitored for safety and will undergo another biopsy at week 12 in Part 2 to assess the expression and biological markers, in addition to longer-term assessments of functional outcomes.

 

Doug Ingram, president and chief executive officer, Sarepta – “Study 102 reinforces our confidence in the potentially transformative benefits of SRP-9001, including among other things, the fact that in the Study’s pre-specified analysis, the participants in the 4-5 age group robustly achieved a statistically significant and clinically meaningful improvement in NSAA over placebo, as predicted by our prior Study 101. For the entire population, while we saw separation at every time point between the active and placebo cohorts, Study 102 did not achieve statistical significance on the primary functional endpoint. In this regard, we are very disappointed that the randomization process resulted in a significant imbalance in baseline NSAA scores between the active and placebo cohorts of the participants ages 6-7, making the 6-7 age groups non-comparable and likely substantially contributing to the inability to achieve statistical significance. Study 102 remains blinded and we will analyze the functional results for all patients, including cross-over participants, once they have achieved the 48-week timepoint in Part 2. We have already enrolled and dosed 11 participants in Study 103, using our commercial process material, and we will have biomarker and safety results from that cohort in the second quarter. And very importantly, Study 102 has provided us with a wealth of information and insight which we will use to refine and complete the protocol for our upcoming trial using commercial process material. We intend to continue to move forward with diligence and urgency to generate the evidence necessary to bring SRP-9001 to waiting Duchenne patients around the world.”

 

*The NSAA is a 17-item rating scale used to measure functional motor abilities in ambulant children with Duchenne. It is used to monitor the disease’s progression and treatment effects, which makes it suitable as an endpoint in clinical trials for Duchenne.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a rare, fatal neuromuscular genetic disease in approximately one in every 3,500-5,000 males worldwide. DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. Symptoms of DMD usually appear in infants and toddlers. Affected children may experience developmental delays such as difficulty in walking, climbing stairs or standing from a sitting position. As the disease progresses, muscle weakness in the lower limbs spreads to the arms, neck and other areas. Most patients require full-time use of a wheelchair in their early teens and then progressively lose the ability to independently perform daily living activities such as using the restroom, bathing and feeding. Eventually, increasing breathing difficulty due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and patients usually succumb to the disease in their twenties.

About SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)

SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. In December 2019, the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital.

About Sarepta

Sarepta Therapeutics, Inc., a biopharmaceutical company, is working to unlock the potential of RNA-based and gene therapy technologies to treat serious and life-threatening diseases like Duchenne muscular dystrophy (DMD). Sarepta’s primary focus is to advance new treatments for DMD rapidly.

More interesting links

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The power of giving back

Giving Tuesday – the global day of giving

Join us for the 8th annual Giving Tuesday on December 1rst, 2020!

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 

 

What was achieved in 2020?

The seventh annual GivingTuesday in Canada exceeded all expectations. Over 7,000 partners came together, and millions of Canadians joined in, creating national awareness, recognition and a massive impact on donations (+ 1247% at CanadaHelps since 2012) and other forms of giving.

 

What is GivingTuesday?

After Black Friday and Cyber Monday … imagine a day dedicated to giving back, around the world, across Canada and in our community. Black Friday kicks off the holiday shopping season; GivingTuesday ( December 1st, 2020) marks the giving season’s opening day. Visit www.givingtuesday.ca for more information.

 

Who is involved?

In Canada, GivingTuesday is an initiative of CanadaHelps and The GIV3 Foundation and 15 founding partners. They have been joined by thousands of Canadian charities, businesses and communities in a collective call to all Canadians to support charities of their choice. GivingTuesday was started in 2012 in the USA by the UN Foundation and the 92nd Street Y. The movement now includes more than 150 countries.

 

2019 Highlights

“Around the world, #GivingTuesday is a grassroots movement that illustrates the impact we can have when we all pull together. The investments we make—in both time and funding—can help solve the urgent problems of today and will make the next generation stronger. “ – President Barack Obama

 

Help someone afflicted with 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 muscle 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 people afflicted with DMD. Our organization raises funds for promising research and raises awareness to have a unified DMD community across Canada to quickly access new treatment.

 

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: providing access to new treatments as fast as possible and participating 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

 

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.

World Duchenne Awareness Day

September 7 is World Duchenne Awareness Day. On this day, we raise awareness for Duchenne and Becker muscular dystrophy (DMD and BMD) around the globe.

An initiative coordinated by the World Duchenne Organization

Together, we are stronger.

The current Corona pandemic is profoundly impacting our daily lives. It changes the way we spend time altering our habits. Although it is changing our everyday life, it cannot overrule our projects and voices.

As the Duchenne community, we are very aware of how strongly this affects us in terms of isolation, facing emergencies and adaptation. We are familiar with words as fear, pain and distance. However, World Duchenne Organization is trying to turn fear into hope, pain into resilience, and distance into closeness.

This World Duchenne Awareness Day 2020, we share a message that many people have experienced and are still experiencing: Together, we are stronger. Even after the emergency will finish, our cause will always be there, our engagement is still there, and we need every single person to be involved.

 

Duchenne and the brain

September 7 is World Duchenne Awareness Day. On this day, we raise awareness for Duchenne and Becker muscular dystrophy (DMD and BMD) around the globe. As every year, we have a special theme that deserves more attention: this year, that will be Duchenne and the brain. The seventh day of the ninth month represents the 79 exons in the dystrophin gene.

The same protein that is missing in the muscle, causing muscle breakdown, is also missing in the brain. This can cause problems like learning difficulties and behavioural issues such as ADD, ADHD, OCD and autism. For many families, these issues result in more stress and worries in daily life than physical problems.

This crucial neurological aspect of DMD/BMD was already recognized in 1861 when Duchenne de Boulogne first described this neuromuscular condition. In the last decades, however, most of the efforts have focused on improving outcomes related to muscle weakness. Brain involvement received less attention.

What we plea for is early screening, appropriate testing, more research and better care on this aspect of the disease. This is needed to have everyone reach their full potential.

 

Video from World Duchenne Organization

 

About DMD/BMD

Duchenne and Becker muscular dystrophy (DMD and BMD) are two muscle-wasting conditions. Mutations in the dystrophin gene cause both. A certain part of the DNA is missing, duplicated, or changed, so the code cannot be read correctly by the body. This leads to the absence of the dystrophin protein, which plays an essential role in the muscle and brain. Without dystrophin, muscle cells easily get damaged, leading to a loss of these cells and, thereby, muscle function.

This can also happen in the brain, where dystrophin is missing as well. Where Duchenne individuals have a complete lack of dystrophin, people affected with Becker muscular dystrophy have lower levels or a shorter version of this protein.

 

Key facts

  • Duchenne and Becker muscular dystrophy are rare genetic diseases defined by muscle weakness
  • The dystrophin protein cannot be made due to an error on the X chromosome
  • Currently, there is no cure available for this fatal disease
  • Each year, 1 in 5.000 newborn boys receive the diagnosis DMD
  • Lack of awareness contributes to an average delay of diagnosis of 2,5 years

 

Useful links

Sarepta signs agreement with Hansa Biopharma for imlifidase

Sarepta Therapeutics signs agreement with Hansa Biopharma for imlifidase. This exclusive worldwide license enables Sarepta to develop and promote imlifidase as a pre-treatment for gene therapy in Duchenne and Limb-girdle muscular dystrophy patients who have pre-existing antibodies to AAV.

Original press release > CAMBRIDGE, Mass., July 02, 2020 (GLOBE NEWSWIRE)

 

Sarepta Therapeutics, Inc. announced an agreement with Hansa Biopharma, the leader in immunomodulatory enzyme technology for rare Immunoglobulin G (IgG) mediated diseases, for imlifidase. Under the terms of the agreement, Sarepta obtains an exclusive, worldwide license to develop and promote imlifidase as a pre-treatment to enable Sarepta gene therapy administration in Duchenne muscular dystrophy (DMD) and Limb-girdle muscular dystrophy (LGMD), for patients who may otherwise not be eligible for treatment.

Sarepta’s investigational gene therapies use an adeno-associated virus (AAV), and patients with Duchenne and LGMD who have pre-existing IgG antibodies are not currently eligible for treatment with any AAV-based gene therapies. Imlifidase is an antibody-cleaving enzyme that targets explicitly IgG and inhibits an IgG-mediated immune response. Imlifidase has a rapid onset of action, cleaving IgG-antibodies and inhibiting their reactivity within hours after administration, thus clearing the AAV-IgG antibodies that would typically preclude dosing or re-dosing with AAV.

 

 

Doug Ingram, president and chief executive officer, Sarepta Therapeutics – “As we expand our leadership position in genetic medicine and build out our gene therapy engine, one of Sarepta’s central ambitions is to find scientific solutions that bring our potentially life-saving therapies to the greatest number of the rare disease patients we serve. One of the current limitations of gene therapy is the inability to treat patients who have pre-existing neutralizing antibodies to the AAV vector. While our AAVrh74 vector has been associated with a low screen out rate for neutralizing antibodies, even that low rate is inconsistent with our mission. In pre-clinical and clinical models, Hansa’s technology has shown the ability to clear the IgG antibodies that prevent dosing AAV-based gene therapies. If successful, this could offer the potential of extending our gene therapy treatments to DMD and LGMD patients who would otherwise have been denied access due to pre-existing antibodies.”

 

 

Søren Tulstrup, president and chief executive officer, Hansa Biopharma – “We see significant potential for our enzyme technology in the gene therapy space overall, and we are excited to partner with Sarepta, a leading player in the field, to use the unique features of imlifidase to potentially enable gene therapy treatment in patients who today aren’t eligible for these breakthrough therapies due to pre-existing neutralizing antibodies in two conditions with a very high unmet medical need.”

 

Under the terms of the agreement, Hansa will receive an upfront payment of $10 million and is eligible for additional development, regulatory and sales milestone payments potentially totalling up to $397.5 million. Hansa will book all sales of imlifidase and will earn tiered royalties up to the mid-teens on any incremental gene therapy sales that arise from treating antibody-positive patients enabled through imlifidase pre-treatment.

Imlifidase

  • Imlifidase is a unique antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets IgG and inhibits IgG-mediated immune response.
  • Using imlifidase is a novel approach to eliminate pathogenic IgG. It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their reactivity within hours after administration.
  • Hansa is leveraging its enzyme technology platform to develop immunomodulating treatments for rare IgG-mediated autoimmune conditions, transplant rejection and cancer.
  • The Research and Development program is advancing the unique enzyme technology to develop the next generation IgG-cleaving enzymes with lower immunogenicity, for repeat dosing. Imlifidase has a strong patent portfolio offering protection beyond 2035.

 

Sarepta Therapeutics

Sarepta Therapeutics, Inc., a biopharmaceutical company, is working to unlock the potential of RNA-based and gene therapy technologies for the treatment of severe and life-threatening diseases like Duchenne muscular dystrophy (DMD). Sarepta’s primary focus is to advance new therapies for DMD rapidly. Learn more here.

Sources

  • Sarepta Therapeutics, Inc.
  • Hansa Biopharma
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COVID-19 for people affected by Duchenne or Becker

WHAT WE KNOW ABOUT COVID-19

What do we know about COVID-19 for people affected by Duchenne or Becker? Due to the increasing concerns regarding the COVID-19 virus for people with Duchenne and Becker muscular dystrophy, the World Duchenne Organization had hosted a webinar last Saturday for its members.  All these information are provided by World Duchenne Organization

General information

  • It’s a respiratory virus that can be spread by aerosols: little droplets when you cough or sneeze.
  • Symptoms are coughing, having fever, shortness of breath and difficulty breathing.
  • The virus can survive for hours on hard surfaces, so you don’t have to see the person who is symptomatic and spreading.
  • The highest risks are the older population above 60 and vulnerable people.
  • This Wednesday, the WHO officially declared COVID-19 a pandemic.

Stanford University School of Medicine Webinar ‘Coronavirus for non-virologists

And DMD/BMD

  • There is no expertise about Duchenne / Becker muscular dystrophy and the coronavirus as we don’t know any DMD/BMD patient affected by it
  • We have asked DMD experts to give a reaction to the questions of our families
  • Situation and national rules will be different in all countries

 

How to follow instructions if resources are scarce?

Prof. Dr. Jonathan Finder –“The best way to protect your sons is the avoidance of crowds and careful handwashing with soap and water.”

Prof. Dr. Nathalie Goemans –“We cannot stress enough the rules of common sense and hygiene, applicable to the general population and even more important for the helpers and caregivers.”

Elizabeth Vroom –“Next to washing your hands often, it’s necessary to clean surfaces, door handles and touch screens regularly.”

 

What impacts do steroids have on the immune system?

Prof. Dr. Jonathan Finder –“Steroids are a mild immunosuppressant and reduce the activity of lymphocytes, and these are the cells that help fight off viruses.”

 

Do people DMD/BMD patients have a higher chance of catching the virus?

Prof. Dr. Jonathan Finder – “No, and possibly they have a lower risk given that they are less likely to be touching doorknobs and handles and shake hands and the like. Those in schools or just out and about have the same risk from respiratory droplets.”

Prof. Dr. Nathalie Goemans – “We cannot stress enough that containing this epidemic is everyone’s responsibility, we should all temporarily restrict our contacts and stay as much as possible at home, respecting strict measures of hygiene.”

 

When infected, will it take them longer to fight it off?

Prof. Dr. Jonathan Finder –“We have no information about this. Presuming that steroids are being used, it is likely that the illness will be a bit harder to fight since steroids are mildly immunosuppressant. This is NOT to say that one should stop steroids: DO NOT STOP STEROIDS, as this is dangerous and riskier than the possible risks of COVID-19.”

Prof. Dr. Nathalie Goemans –“Yes, it is known that a severe course of COVID-19 can cause permanent damage to the lungs. On a positive note: although steroids are known to reduce immunity, it might well be that steroids could have a protective role in the pathophysiology (cytokine-storm) of severe ARDS in COVID-19 but we don’t know yet.”

 

Are they at higher-risk or ‘vulnerable people’ most likely to die?

Prof. Dr. Jonathan Finder – “They are at higher risk to be sure as the illness is a viral pneumonia, and having pneumonia is a risk for respiratory failure in this population. But as for “more likely to die” I would say NO as these patients are younger and for the most part do not have underlying lung disease. Those with chronic lung disease are the highest risk group, along with the elderly.

On the other hand, cardiac disease is a risk factor, and there is a great deal of cardiac disease in the DMD population. Thus I do have concerns about the risk of COVID-19 infection for those patients with heart failure.”

 

More answer here < WDO Webinar: COVID 19 and Duchenne & Becker muscular dystrophy >

Defeat Duchenne Family Forum with Jacques P. Tremblay

Key Note Speaker: Dr. Jacques P. Tremblay

Presented by PTC Therapeutics and in partnership with Jesse’s Journey and La Force DMD, this unique educational event provides the opportunity for families and caregivers navigating the Duchenne journey, to come together with researchers, like Jacques P. Tremblay, clinicians, and industry professionals for a day of education and inspiration.

Our Key Note Speaker will be Dr. Jacques P. Tremblay

Dr. Jacques P. Tremblay received a Ph.D. in Neuroscience from the University of California at San Diego in 1974. Since that time, he has been at Laval University as a postdoctoral researcher, Professor and Director of the Department of Anatomy. He is currently a full Professor in the Department of Molecular Medicine. He has worked specifically on myoblast transplantation* as a treatment for Duchenne muscular dystrophy. He has conducted a Phase I clinical trial of that potential therapy and he is currently conducting with Dr. Craig Campbell a Phase I/II clinical trial of that therapeutic approach. For the last 3 years, he also worked on gene correction with the CRISPR/Cas9 technology* for Duchenne muscular dystrophy, Friedreich’s Ataxia and Familial Alzheimer’s disease. This new exciting technology permits to correct mutations responsible for many hereditary diseases.

Presentation

Dr. Tremblay will provide an overview of the basics of genes, messenger RNA and proteins and build a gradual understanding of how mutations in the dystrophin gene lead to Duchenne or Becker muscular dystrophy. He will then present the different therapeutic approaches currently investigated around the world: prednisone, deflazacort, myoblast transplantation, exon skipping, micro-dystrophin and correction of the dystrophin gene with the new CRISPR/Cas9 technology.

*Transplanting myoblasts

Consists of transplanting healthy muscle cells, called myoblasts, into forearm muscle in Duchenne patients and measuring muscle strength at 3 and six months posttransplantation. This trial is for patients aged 16 and over.

*CRISPR / Cas9 technology

CRISPR/cas9 technology is a technology that was first identified in bacteria. Bacteria were using this to cut up the genome of the viruses that were infecting them. About five years ago, researchers noticed that this technology allows not only to cut up virus genes but that it can also cut genes in animals, plants and especially in humans.

RARE DISEASE DAY – February 29, 2020

February 29, 2020, will be the thirteenth international Rare Disease Day coordinated by EURORDIS. On and around this day hundreds of patient organizations from countries and regions all over the world will hold awareness-raising activities. 

What is the rare disease day?

The first Rare Disease Day was celebrated in 2008 on 29 February, a ‘rare’ date that happens only once every four years. Ever since then, Rare Disease Day has taken place on the last day of February, this month is known for having a ‘rare’ number of days. Rare Disease Day is the opportunity advocate for rare diseases as a human rights priority at local, national and international levels for a more inclusive society.

Rare Disease Day takes place on the last day of February each year. The primary objective of Rare Disease Day is to raise awareness among the general public and decision-makers about rare diseases and their impact on patients’ lives. 

Building awareness of rare diseases is important because 1 in 20 people will live with a rare disease at some point in their life. There is no cure for the majority of rare diseases, and many go undiagnosed. Rare Disease Day improves knowledge amongst the general public of rare diseases while encouraging researchers and decision-makers to address the needs of those living with rare diseases.

Because it manifests itself only rarely, Duchenne muscular dystrophy (DMD) falls within the rare diseases category.  Learn about our history here.

Raising awareness of what it means to be rare. There are over 300 million people worldwide living with a rare disease. Together across borders, and across the 6000+ rare diseases we need more equitable access to diagnosis, treatment, care and social opportunity.

 

Rare is many worldwide Rare is strong every dayRare is proud everywhere

 

Statistics and facts

  • 300 million people living with a rare disease worldwide
  • Over 6000 different rare diseases
  • 72% of rare diseases are genetic
  • 70% of those genetic rare diseases start in childhood
  • Rare diseases currently affect 3.5% – 5.9% of the worldwide population.
  • If all of the people with rare diseases lived in one country, it would be the world’s 3rd most populous country. In Quebec, it is estimating that close to one in 20 people will be affected or have a rare disease, which means nearly 500,000 Quebeckers.
  • 30% of children with rare disease will not live to see their 5th birthday
  • Rare diseases are responsible for 35% of deaths in the first year of life.
  • Only 5 percent of rare diseases have treatments. Drug research that helps a limited number of people can be cost-prohibitive for pharmaceutical companies.

 

Follow Rare Disease Day

Stay up to date with the latest news from this year’s Rare Disease Day campaign by following them on Facebook, Twitter and Instagram.

 

Finding a support group is important

A rare disease can be isolating for the patient as well as for the caregiver, especially when it’s your child who has the condition. Connecting with others can be essential, not only for support but also to share information and resources.

The Quebec Coalition for Orphan Diseases (RQMO) works to provide information and support to patients, their families, and healthcare professionals. Their website is full of relevant information both for professionals and caregivers. These rare diseases are often of little interest to researchers and organizations who fund research. The RQMO aims to advance knowledge about the various rare and orphan diseases by promoting exchanges between patients and researchers.

The Canadian Organization for Rare Disorders (CORD) is Canada’s national network for organizations representing all those with rare disorders. CORD provides a strong collective voice to advocate for health policy and a healthcare system. CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment, and services for all rare disorders in Canada.

February 29th is the Rare Disease Day to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives.

 

We wish to emphasize that the information contained in this article comes mainly from the RQMO website, the CORD website and the Rare Disease Day website.

We thank them.

DEFEAT DUCHENNE FAMILY FORUM – MONTREAL

We are excited to share that the next Defeat Duchenne Family Forum will take place in Montréal, Québec, on Saturday, May 2, 2020.

Presented by PTC Therapeutics and in partnership with Jesse’s Journey, this unique educational event provides the opportunity for you – families and caregivers navigating the Duchenne journey, to come together with researchers, clinicians, and industry professionals for a day of education and inspiration. More than that, the Defeat Duchenne Family Forum provides a support network. A connection opportunity for you to know that they are not alone and together, we can defeat Duchenne. Registration is free, and the day includes take-away resources and all meals (breakfast, lunch, and snacks). Event page here + Facebook event page here

  • What: Defeat Duchenne Family Forum
  • When: Saturday, May 2, 2020 | 9 a.m. – 4 p.m
  • Followed by: Social & Vendor Fair from 4 – 6 p.m
  • Where: Shriners Hospital for Children (1003 Décarie Blvd, Montréal, Québec H4A 0A9)

Stay tuned as we announce registration and full program details in early 2020.

 

The first-ever Duchenne Family Forum In London, Ontario

May 25, 2019, in London, Ontario

This unique event – tailored to families in Canada affected by Duchenne muscular dystrophy – featured updates about the latest research, clinical trials recruiting in Canada, and more. Read more here.

Defeat Duchenne Family Forum in Calgary, Alberta

November 2, 2019, in Calgary, Alberta

More than 75 Canadians came together for the inaugural Defeat Duchenne Family Forum presented by PTC Therapeutics on Saturday, November 2, 2019, in Calgary, Alberta. Families from across Western Canada affected by Duchenne muscular dystrophy came together with world-renowned researchers, clinicians, and industry professionals for a day of education, inspiration, and hope. Read more here.

More links

Learn more about Jesse’s Journey here.

Watch our video about John Davidson, the godfather of the Canadian Duchenne community here.

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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

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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