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Capricor initiates HOPE-2 clinical trial

One of the objectives of our team is to inform you about new treatments. Here’s the latest news from a recent press released by Capricor which announces the clinical trial HOPE-2. Enjoy reading and thank you for sharing these articles within the DMD community.

CAP-1002 aim to maintain or improve cardiac and skeletal muscle function

Capricor’s lead candidate, CAP-1002, is a cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy (DMD). CAP-1002 is an allogeneic product, meaning that it is manufactured from donor heart tissue and then stored until needed for use. CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and alters the immune system’s activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials.

Status by the FDA

CAP-1002 has been granted orphan drug designation by the FDA for the treatment of DMD.

About the HOPE study

Capricor’s previous clinical trial, the HOPE-Duchenne trial, evaluated the safety and efficacy of a single dose of CAP-1002 in boys and young men with heart disease related to Duchenne muscular dystrophy. It found CAP-1002 was safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function.

HOPE 2

Participants in the HOPE-2 trial will be randomized to receive either placebo or CAP-1002, delivered intravenously every three months for a total of 4 administrations. Participants will be followed for a yearlong period following randomization, and an open-label extension (OLE) study is planned. The trial evidence should suggest an appropriate risk/benefit profile of CAP-1002 in the medical indication. Capricor – HOPE-2

Where is the trial?

The medical center in Sacramento is the first site in the nation to begin enrolling and treating participants. Approximately 12 to 15 investigative sites are expected to participate in the trial.

About Capricor

Capricor Therapeutics is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy.

What is DMD?

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,5000. 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.

For more information:

Rare Disease Report

GlobeNews Wire – Press Release

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Edasalonexent: Positive results and no side effects

One of the objectives of our team is to inform you about new treatments. Here’s the latest news from a recent Catabasis press release about edasalonexent. Enjoy reading and thank you for sharing these articles within the DMD community.

The MoveDMD trial through 48 weeks of edasalonexent treatment

 

“ We believe that these effects will ultimately translate to boys with Duchenne maintaining functional abilities longer.”

Said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis, in a press release this week.

The fact that no evidence of side effects or safety has been observed after more than 37 patient-years of exposure to treatment is also encouraging.

What is Edasalonexent?

Edasalonexent (CAT-1004)  is being developed as a potential foundational disease-modifying therapy for all patients affected by DMD, regardless of their underlying mutation. It is an investigational oral small molecule. Edasalonexent inhibits NF-kB, a protein that is activated by DMD and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration.

You can watch our video here to have a more in-depth explanation about the basis of this treatment.

 

Positive results:

Statistically significant improvement was observed compared to the off-treatment control period. These improvements show a slowing of disease progression and are in addition to the improvements found in all assessments of muscle function through more than a year of edasalonexent treatment.

What’s next?

Catabasis is preparing for a Phase 3 trial that will enroll approximately 125 boys with DMD between the ages of 4-7 years old regardless of mutation type and who have not been on steroids for at least six months. It is planned to be a single, global, placebo-controlled Phase 3 trial with two boys receiving edasalonexent for every one boy receiving placebo. After 12 months in the trial, all boys are expected to receive edasalonexent in an open-label extension.

Approbation in the US: FDA Status

The FDA has granted orphan drug, fast track and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results reported to-date, please visit www.catabasis.com.

About DMD

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.

For more information:

More information about the trial:  DMDtrials@catabasis.com and Catabasis -clinical trials

More: Portrait of Duchenne – edasalonexent cat-1004 – La Force DMD

Press release: www.catabasis.com

Business Wire: www.businesswire.com

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A clinical trial on utrophin modulation is completed

One of the objectives of our team is to inform you about new treatments. You can catch up on the last news in the latest press release by Summit Therapeutics company about ezutromid, a utrophin modulation. We wish you a happy reading and thank you for sharing those articles within the DMD community.

“PhaseOutDMD” clinical trial for ezutromid is completed

“We believe the early improvements seen in muscle health in the interim data from PhaseOut DMD indicate ezutromid is reducing DMD disease severity. In the full trial results, we aim to see continued utrophin modulation and sustained changes in magnetic resonance parameters. These results, if positive, could form the basis of a regulatory filing of ezutromid, bringing this universal treatment to patients more rapidly.”

Said Dr. David Roblin, Chief Medical Officer and President of R&D of Summit.

What is “PhaseOutDMD”?

PhaseOut DMD is a Phase 2 open-label, multi-centre trial of the Company’s utrophin modulator, ezutromid, in patients with DMD. Previously announced 24-week interim data from PhaseOut DMD showed evidence of activity across three different measures. Specifically, ezutromid:

• Maintained the production of utrophin, a naturally occurring protein that can potentially substitute for dystrophin, as measured by muscle biopsy;

• Significantly and meaningfully reduced muscle damage, as measured by muscle biopsy; and

• Significantly reduced muscle inflammation, as measured by magnetic resonance.

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 simulated 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.

 

This video explains what is utrophin.

About Summit Therapeutics

Summit is a biopharmaceutical company focused on the discovery, development and commercialization of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programmes focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc).

What is DMD?

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,5000. 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.

Useful links

Here are a few additional links including the initial news release of the Summit Therapeutics company.

Link to the news release: www.summitplc.com

Link to additional information on utrophin modulation:

Positive data about utrophin modulator – La Force DMD

Portrait of Duchenne – Utrophin modulator – La Force DMD

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New DMD exon-skipping therapy on the way

Following positive results obtained in Phase I/II trial of SRP-4053 (Golodirsen), Sarepta Therapeutics has announced a plan to submit a new drug application (NDA) for accelerated approval of Golodirsen in patients with Duchenne muscular dystrophy (DMD).

According to results of the clinical study, Golodirsen significantly boosted dystrophin protein production in 25 boys with confirmed deletions of the DMD gene amenable to exon 53 skipping. This mutation affects about 8 percent of all DMD patients.

 

What is Golodirsen?

Golodirsen uses exon-skipping technology and works by binding to exon 53 of the dystrophin sequence to exclude, or skip, this part of the sequence. This helps produce a smaller but functional form of dystrophin protein.

 

Positive results

Golodirsen showed potential to treat Duchenne muscular dystrophy (DMD) in a first clinical trial of DMD patients. Press release

 

Why do we need to skip an exon?

DMD is caused by specific errors (mutations) in the gene that codes for dystrophin. Dystrophin is a protein that plays a crucial role in the function of muscle cells and protects them from damage as muscles contract and relaxes. These mutations in the dystrophin gene lead to a lack of dystrophin protein in muscles. Without enough dystrophin, muscles gradually grow weaker until they can’t move at all, and eventually breathing and heart function are lost.

The condition is universally fatal, and death usually occurs before the age of 30 generally due to respiratory or cardiac failure.

 

More about Golodirsen

Golodirsen uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO)* chemistry and exon-skipping technology to skip exon 53 of the DMD gene. Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for the production of an internally truncated but functional dystrophin protein.

Golodirsen is one of the investigational candidates currently being evaluated in the ESSENCE study, a global, randomized double-blind, placebo-controlled study evaluating efficacy and safety in patients amenable to skipping exons 45 or 53.

*a phosphorodiamidate Morpholino oligomer (PMO), is a type of oligomer molecule (colloquially, an oligo) used in molecular biology to modify gene expression. 

 

More about clinical trial

 ESSENCE: Phase III Study

Purpose: The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53 respectively.

Location: United States, Europe, Canada, Israel

For more information, please visit www.clinicaltrials.gov or www.essencetrial.com

 

Sources:

Clinical Trials

Muscular Dystrophy News

Investor Relations

Wikipedia

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 concerning 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 the 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.

The 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.

 

MAKE SURE NOT TO MISS OUR SHORT DOCS SERIES

SUSCRIBE TO OUR NEWSLETTER. HERE.

 

 

 

 

 

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

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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’S UNBELIEVABLE GIFT TO HELP HIS PATIENT

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.

MAKE A DONATION:  BRUCE’S CAMPAIGN