Microdystrophin gene transfer trial on Hold

Solid Biosciences Announces Clinical Hold On SGT-001 microdystrophin gene transfer Clinical Phase I/II Clinical Trial for Duchenne Muscular Dystrophy. 

Solid Biosciences Inc. announced it had received notification from the U.S. Food and Drug Administration (FDA) that IGNITE DMD, its Phase I/II clinical trial for microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical Hold.

What happened?

The first patient dosed in the clinical trial was a non-ambulatory adolescent. Several days after administration the patient was hospitalized due to laboratory findings that included a decrease in platelet count followed by a reduction in red blood cell count and evidence of complement activation. *The complement system is an enzyme cascade that helps defend against infection. 

How is the patient now?

The patient was admitted to the hospital, received treatment, and he is home with his family with no symptoms.

What is a clinical hold?

Solid reported the event to the FDA and, because it was unexpected, classified it as a Suspected Unexpected Serious Adverse Reaction (SUSAR).

What’s next?

The team at Solid will be working with the principal investigator and FDA to fully understand the cause and nature of this event, as well as identify appropriate next steps as soon as possible.


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 the clinical trial:

The Phase I/II clinical trial, called IGNITE DMD, is a randomized, controlled, open-label, single ascending dose study that will evaluate the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients with DMD.  IGNITE DMD, adaptive in nature, will allow Solid Biosciences to adjust dose and number of patients as the study progresses to efficiently characterize the safety and efficacy of SGT-001. The patient screening will begin at their first participating study in one location in the United States in the coming days. Solid Biosciences is working to bring on additional sites in the United States and abroad.

About SGT-001

SGT-001 is a novel adeno-associated virus* (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD. 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.


  • Clinical trial: Here
  • Letter to the Duchenne Community About the Status of the IGNITE DMD Clinical Trial: Here
  • Link to press release: Here
  • More info on our previous blog post: Here

Decision from the FDA’s Office for Ataluren

Today, PTC Therapeutics, Inc, announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA’s prior position and denied PTC’s appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren. In its letter, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway.

This would involve a re-submission of an NDA containing the current data on effectiveness of ataluren with new data to be generated on dystrophin production in nonsense mutation Duchenne muscular dystrophy (nmDMD) patients’ muscles, as quantified by procedures to be agreed upon between PTC and the FDA and using newer technologies. The letter adds that PTC’s Study 041, which is currently enrolling, could serve as the confirmatory post-approval trial required in connection with the accelerated approval framework. In a clarification teleconference with the FDA promptly after receiving the letter, PTC indicated its intent to follow the FDA’s recommendation and preliminarily discussed methods to collect such dystrophin data and expedite this potential path forward.

Patient will still have access to treatment:

Based on these interactions, PTC currently intends to maintain patients in the U.S. currently receiving ataluren for nmDMD through an expanded access clinical program during this process.

About the AdCom watch our video:


About Translarna™ (ataluren)

This treatment is the first to target an underlying genetic cause of DMD: a nonsense mutation (nmDMD). It aims to slow the progression of DMD by restoring the function of a specific protein, dystrophin. In DMD, a nonsense mutation corrupts the genetic machinery that builds a working version of this protein, which is essential for proper neuromuscular function.

In clinical trials, ataluren has shown some benefit in slowing the loss of motor skills in some children with nmDMD. Some boys treated with ataluren could more easily undertake a range of physical activities, including walking, climbing and descending stairs and other motor functions.

In 2014, the European Medicines Agency granted Translarna™ (ataluren) conditional marketing authorization to treat nmDMD in children of 5 years of age or more and still able to walk. Translarna™ (ataluren) is also approved for use in South Korea and Israel.

At the end of 2016, Translarna™ (ataluren) received marketing authorization by the European Medicines Agency and is available in Europe and regions that reference that authorization.

On the 25h of October, the Office of Drug Evaluation I of the FDA provided a Complete Response Letter (CRL) for the application to market ataluren in the United States. This means that the FDA believes ataluren cannot be approved at this time. PTC Therapeutics Inc. is planning on filing a formal dispute resolution request next week. The dispute resolution process gives PTC an opportunity to appeal the letter issued by the Neurology division and have the decision on the application reviewed by the Office of New Products within the FDA. PTC Press Release


Understanding Ataluren approbation process

For more information

What is the AdCom?
Families at the AdCom September, 2017
FDA close the door on Ataluren

Short Docs series: Portrait of Duchenne Canada

Our web short documentary series, “Portrait of Duchenne Canada”, will be coming son! 

We will present the experiences of families and children with DMD across Canada.What’s it like to live with Duchenne muscular dystrophy (DMD)? As a parent, to face the reality that your child has a fatal disease? As a person with DMD, to face an inevitable, premature death in your twenties? As a sibling, relative or friend, to face mourning your loss?


The journey is beyond words.

Our web short documentary series, “Portrait of Duchenne Canada”, will present the experiences of families and children with DMD. You will meet five Canadian families who are taking on the challenges of raising money and leading the way in advocating for access to new treatments. New treatments are on the horizon and families need to be heard to advocate for ways to access them quickly.

Today, more than ever, it’s time to share their stories.


Join the fight!

Duchenne muscular dystrophy (DMD) is a rare disease with no cure. Orphan drugs that target its causes are being developed now. But we need your help. Research is the only hope. And research needs funding.For the past 20 years, Canadian families in the DMD community have been raising funds for and awareness of DMD. Their actions and successes have funded current research. Now that new treatments are emerging, families need to unite and be heard in order to access new treatments quickly.

Our part is to tell their story. Sharing these stories is one step forward towards the cure.









February is the Rare Disease Month

February is the Rare Disease Month. Because it manifests itself only rarely, Duchenne muscular dystrophy (DMD) falls within the rare diseases category. It is also a treatment orphan disease.

Since this is February, it is pertinent for us to tell you about it. 


A few distinctions …

  • A rare disease is defined as a disease that affects less than 1 in 2000 (DMD affects 1 in 3500, which makes it a rare disease).
  • The RQMO estimates that, in Québec, nearly one in 20 is afflicted by or carrier of a rare disease, for a total of nearly 500,000 Quebecers. Many rare diseases are chronic, progressive and fatal. The CORD estimates approximately, 3 million Canadians and their families face a debilitating disease that severely impacts their lives.
  • Nearly 75% of these diseases affect children, and about 80% of them are genetic.
  • The term “orphan disease” is often confused with the term “rare disease” because the vast majority of rare diseases are orphans in many ways.
  • A medical condition is referred to as an orphan disease if there is no treatment for the disease other than treating the symptoms, as is the case for DMD: for example, prednisone is a medication that has an effect on the symptoms but not on the cause of the disease.


The rarity of these diseases creates obstacles and needs for afflicted people both in our health system and in society in general.

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. Being rare, these 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 that works for those with rare disorders. CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment, and services for all rare disorders in Canada.

February 28Th 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 come mainly from the RQMO website and the CORD website.

We thank them.


Rare disease day

Make a donation to La Force


Positive data about utrophin modulator


Today, Summit Therapeutics announced positive 24-week interim data from PhaseOut DMD (Phase 2 clinical trial of the utrophin modulator ezutromid). This data showed a significant reduction in muscle damage and an increase in utrophin in muscle biopsies. The company plans to hold a webinar with the community. Below you’ll find some FAQs and the press release.


What does this data mean?

  • Ezutromid treatment led to a significant reduction in muscle damage and increased production of utrophin in muscle fibres;
  • Utrophin modulation maintains utrophin production in mature muscle fibres, enabling utrophin to replace the need for dystrophin in DMD muscles;
  • Ezutromid has been well tolerated to date in all patients participating in PhaseOut DMD;


Will there be another clinical trial? When will it start/where will it take place?

  • Summit Therapeutics expects to conduct another clinical trial aimed at getting regulatory approval for ezutromid to be marketed in the US and Europe;
  • Summit Therapeutics is actively planning the next trial and expects to provide a timeline for the start of that trial once they have the 48-week data;
  • It is expected to be a global trial, and the participating countries and sites will be announced closer to the initiation of the trial;


When will ezutromid be available on the market?

  • Summit Therapeutics is awaiting the 48-week data from PhaseOut DMD before finalizing their plans for the next trial:


What is exactly utrophin?

You can watch our interview with Michelle Avery, the Director of Investor Relations for Summit Therapeutics, to know more about it:




What is utrophin?

The human body naturally produces utrophin, a protein, when a muscle is first forming or when a muscle is repairing. As a muscle matures, dystrophin replaces utrophin. However, in people with Duchenne muscular dystrophy (DMD), dystrophin does not function properly.

Utrophin is functionally and structurally similar to dystrophin. Preclinical trials that have stimulated sustained utrophin production have shown that it could potentially replace dystrophin in people with Duchenne muscular dystrophy (DMD). The replacement of dysfunctional dystrophin with functional utrophin might have a highly positive impact on muscle performance.

Summit Therapeutics believes that utrophin may slow or even stop the progression of DMD.

More information: PPMD  –  Wikipedia  – NCBI


At this moment the only way to access the treatment is in the clinical trials.

About PhaseOut DMD Clinical trials: http://www.utrophintrials.com

Link to press release: http://otp.investis.com/clients/uk/summit_corporation_plc/rns/regulatory-story.aspx?cid=1575&newsid=970514

More info on our previous blog post: https://laforcedmd.com/utrophin/


Bruce Babington, an Osteopath, based in Ste-Agathe-des-Monts, Québec, is taking on the challenge of cycling across Canada in July 2018. He was inspired by his patient, Anakin Lacasse, who is afflicted with Duchenne muscular dystrophy. Why riding across Canada? To spread knowledge about DMD and raise money for research, the only hope for children like Anakin…

Riding across Canada for hope!

Bruce plans to cycle an average of 200 KM from Mont-Tremblant to Vancouver and complete the journey in 24 days. When he first started to treat Anakin, he didn’t know much about DMD, but it became clear to him very fast that there was no quick fix! He promptly made the decision offer him free services for life. He also had the idea in the back of his mind that someday he would like to go across Canada by bike. He also learned about Fondation La Force, the organization inspired by Anakin, which helps him and other people afflicted with Duchenne to get closer to a cure. At one point, the idea of cycling across Canada for La Force crossed his mind, and he made the decision. He will be taking one month off in July of 2018, with the goal of cycling 200 KM a day and complete the distance between Mt-Tremblant and Vancouver within 24 days.


Go big or go home!

“You know, the challenge I’ll face in that 24-day period is nothing like the challenge Anakin is going to face every single day of his life. What I can really do for him is try as much as I can to help promote La Fondation La Force and really spread the knowledge of what Duchenne really is” said Bruce Babington to our team.


About Duchenne Muscular Dystrophy (DMD)

A degenerative disease of the muscles for which there is no treatment

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families. The typical evolution of the disease inevitably brings about premature death between the ages of 18 and 25.


We need your donation:

We have an ongoing campaign to finance Bruce’s ride. In 2018, our team will work hard to give the visibility Bruce needs to unite people around DMD and raise awareness in the general public about the disease.

Thank you in advance to all of our friends in the DMD community, for encouraging Bruce!


Don’t forget, sharing our stories is also an excellent way to support our cause.




Solid Biosciences Initiates Clinical Trial for Gene Transfer

Last week we had the privilege to meet in person with the team at their headquarter in Boston. They presented their very robust preclinical trial data suggesting that SGT-001, has the potential to slow or stop the progression of DMD, regardless of genetic mutation or disease stage. Now they are announcing the initiating of SGT-001, their investigational microdystrophin gene transfer for DMD and we are so excited as this might become a life-changing treatment for people with DMD.


SGT-001 Clinical trial is an Incredible milestone

This is an incredible milestone for this startup company of four years old. This accomplishment is the reflection of years of ongoing collaboration with the top experts in gene therapy. SGT-001 is based on pioneering research in dystrophin biology by Dr. Dongsheng Duan of the University of Missouri and Dr. Jeffrey Chamberlain of the University of Washington.


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



About the clinical trial:

The Phase I/II clinical trial, called IGNITE DMD, is a randomized, controlled, open-label, single ascending dose study that will evaluate the safety and efficacy of SGT-001 in both ambulatory and non-ambulatory patients with DMD.  IGNITE DMD, adaptive in nature, will allow Solid Biosciences to adjust dose and number of patients as the study progresses to efficiently characterize the safety and efficacy of SGT-001. The patient screening will begin at their first participating study in one location in the United States in the coming days. Solid Biosciences is working to bring on additional sites in the United States and abroad.


About SGT-001

SGT-001 is a novel adeno-associated virus* (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD. 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.

  • Adeno-associated virus > is a small virus which infects humans and some other primate species. Link

  • Systemically administered > Systemic forms of administration affect the whole body (in general).


To know more

Open letter de Solid Biosciences

 If you are interested in learning more about this trial (IGNITE DMD), further information will be available on clinicaltrials.gov shortly.

Gene transfer has the potential to address the genetic cause of Duchenne muscular dystrophy.

Press release

Feeling bad about the holidays shopping frenzy?

Giving Tuesday is your opportunity to give back… This is a day dedicated to generosity just as Black Friday and Cyber Monday are dedicated to shopping. Contributing to our cause will help someone afflicted Duchenne muscular dystrophy (DMD). It’s the moment in Canada and the whole world to celebrate giving back and supporting causes that matter to you. We are part of the 3,200, charities and organization celebrating this day in Canada.


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 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 raise funds for promising research and raise awareness to have a unified DMD community across Canada to access new treatment quickly.


Give to La Force here

About GivingTuesday

GivingTuesday was launched in Canada in 2013 by GIV3 and CanadaHelps.org and lists 15 founding partners. It’s a day for the entire charitable sector and encourages all Canadians to join the movement and give and volunteer for charities of their choice. GivingTuesday was initially founded in 2012 in the US by 92Y and the UN Foundation.

How to contribute to our cause:

There is no small amount!

Our organization aim 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.


Every dollar is taking us closer to find better treatments and the cure.

Give to La Force 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.




2017, November 17th

On a regular basis, team La Force will relay breaking news to the DMD community in News! our latest blog. We provide news highlights to give you quick-and-easy access to the most recent information about what’s happening worldwide in DMD advocacy and, more importantly, advances in therapy and the availability of new treatments. The short news alerts will also appear in our newsletter and, for members of our DMD community, on the Sign-in page.

News! helps you to stay informed, become aware and share what’s happening in the DMD community to bring us closer to a cure.


Be aware. Get involved. Share.


Jesse’s Journey & Max’s Big Ride fund STAT3 inhibitor research

On October 31, 2017, Jesse’s Journey and Max’s Big Ride – two non-profit groups founded by parents of children with Duchenne muscular dystrophy (DMD) – presented $336,000 in funding to Dr. Patrick Gunning, Professor of Chemistry, Canada Research Chair in Medicinal Chemistry  Tier II, and head of the Gunning Group, a research laboratory at the University of Toronto Mississauga.

Max’s Big Ride raised $35,000 for Max’s Big Fellowship, a new Ph.D. fellowship at the Gunning Group. It was awarded to Yasir Raouf, who will study STAT3 inhibitors in DMD. Earlier this year, Jesse’s Journey approved a $300,000 grant to Dr. Gunning to fund an exciting, first-time, preclinical study of STAT3 and other protein inhibitors to treat DMD.

For more information about the study and how you can get involved: read here


Duke University and Sarepta hunt for gene-editing therapy

Muscular Dystrophy News Today reports that Sarepta Therapeutics has teamed up with Dr. Charles Gersbach’s biomedical engineering laboratory at Duke University to develop gene-editing therapies for Duchenne muscular dystrophy (DMD).

Dr. Gersbach is a pioneer in the use of CRISPR/Cas9 technology, a gene-editing tool that can remove abnormal parts of the dystrophin gene. Mutations in this gene cause DMD. Gerbach says that cutting out specific exons (regions that contain the genetic code or “blueprint” for the dystrophin protein) “has the potential to correct a majority of DMD mutations.”

His team has already shown, in mice models of DMD, that removing abnormal regions of the dystrophin gene restores muscle function. The edited gene was able to produce a normally functioning protein that improved muscle strength in mice.

For more information: read here


New clinical trial of exon-51 skipping therapy for DMD

Wave Life Sciences has begun the first clinical trial of WVE-210201, an exon 51-skipping therapy, in patients with Duchenne muscular dystrophy (DMD). The multicenter, double-blinded, placebo-controlled, dose-finding, Phase 1 clinical trial will study which intravenous doses of WVE-210201 are safe and tolerable in a subgroup of patients with DMD. About 13% of patients with DMD have exon 51 mutations in the dystrophin gene.

The study will enroll up to 40 patients between 5 and 18 years of age, either able or unable to walk. It will begin in the USA, then expand to Europe and other regions. Results are expected in late 2018.

WVE-210201 is an antisense oligonucleotide that throws a cog in the wheels of protein manufacturing. It forces the cellular machinery to skip over a damaged exon – a specific section of genetic code – then resume regular “reading” of the protein’s “blueprint.” The exon 51-skipping process produces an incomplete but functional dystrophin protein, which may restore some muscle function in patients with this mutation.

For more information: read here


Ride for a cure with Dystrospin

On Sunday, November 19, from 7 am to 5 pm, Dystrospin and La Force invite you to spin for Duchenne Muscular Dystrophy. Since 2016, La Fondation La Force has been associated with this event, organized by the Morand family for over five years. All proceeds from the Dystrospin will be donated to two researchers based here in Québec. They are Dr. Jacques Tremblay for his research on CRISPR-Cas9 and Dr. Jérôme Frenette for his research on Osteoprotegerin.


For the love of his son…

Jean-Philippe Morand, whose 11-year-old son, Victor, has DMD, will be directing 10 consecutive spinning sessions. The bravest participants will have the opportunity to pedal 10 in a row.


An event for everyone

Whether you’re a beginner or experienced, it’s sure to be a pleasurable experience, and above all, a unique chance to test your limits. On site, volunteers will help you adjust your bike and explain the training instructions. For the young ones, daycare service will be offered free of charge for the duration of your participation in the event. Inflatable games, drawing, ball games, etc.: you can bet they’ll have a good time! The little ones are also more than welcome.


Every action counts

Duchenne muscular dystrophy is a degenerative disease of the muscles that mainly affects young boys and is 100% fatal. The progression of the disease is unforgiving: the body’s muscles weaken little by little, leaving boys in a wheelchair at the age of 12, with a life expectancy of 20 to 30 years. Nowadays, there is hope through research, and several new treatments are on the horizon. This hope motivates Jean-Philippe to exceed his limits for his son. Every gesture made to raise awareness for the disease will help us fund research and access to new treatments.


How to participate

The activity will take place at PerformanceC2. The Centre is located at Place St-Nicolas, 845, Marie-Victorin in St-Nicolas. Since the number of stationary bikes is limited, you must reserve your bike at for a time that suits you.

Registration and online payment on our highly secure site can be found here> https://www.jedonneenligne.org/fondationchudequebec/frm_detail.php?FrmUID=134


If you can’t participate in this activity? You can still donate here


Learn more: