Our primary goal is to support researchers in finding solutions for patients.

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Over the last 20 years, we have continuously pushed the boundaries of DMD research, funding projects in universities, hospitals, startups, and international collaborations with over €50 million in grantmaking, with a notable surge in recent years. This trend will continue and amplify.

The AMM-Only Project’s support for research has helped advance therapeutic avenues and brought new developments to the forefront of DMD treatment. Now, with growing resources, we continuously strive to strengthen our impact by not only increasing the scale of our funding but also expanding the scope of the projects we support.

Our grantmaking approach is rooted in 3 key values:

  • Agility: We are flexible and responsive, ensuring we can fund emerging opportunities in DMD research quickly and effectively.
  • Impact: We prioritize groundbreaking research that challenges conventional approaches and drives advances in DMD therapies and patient care.
  • Comprehensive Support: We provide funding across the spectrum of DMD research, from foundational science to clinical treatments, ensuring all aspects of the disease are addressed.

There are two global streams of funding in our grantmaking programs.

Stream 01

The first and main category of grantmaking is Scientific and Medical Research, representing 98% of funding.

  • Our flagship project: the startup SQY Therapeutics and the clinical Trial AVANCE 1 of its SQY51 drug.
    SQY Therapeutics, a non-profit biotech (owned by an endowment fund created by Associations), is currently testing its drug candidate SQY51 in the AVANCE1 clinical trial in Garches, France. SQY51 works like a “molecular patch” by skipping over the faulty part of the DMD-causing gene, allowing the body to produce a key muscle-preserving protein. The first phase of the trial showed encouraging results with no major side effects, and the second phase is now underway. SQY Therapeutics has been a significant part of the Association’s spending, with its research supported by Only Watch funds since 2015.

 

  • Our second portfolio concerns grantmaking for fundamental and applied research outside of SQY Therapeutics’ R&D programs including the following grants:

 

    • PhD and Post-Doctoral Grants, also known as the “Paul Pettavino or Philippe Ferreyrolles” Fellowships
      Over the years, our Association has been a flexible and reliable supporter, empowering a new generation of researchers through doctoral and post-doctoral fellowships. We consistently fund PhD and post-doctoral projects in hospitals and universities, aiming to advance knowledge on Duchenne Muscular Dystrophy potential treatment pathways and underlying mechanisms. Each year, we anticipate awarding up to 7 new grantees.
    • Research Grants for Operational Costs on DMD, also referred to as the ‘Adrien-Tanaphum Ferré’ GrantThe Association recognizes that some research projects face significant operational hurdles that can delay their launch or impact their progress. To address this, we have established a grant designed to cover essential operational costs, including funding for labs, equipment, and other critical tools needed.
    • The Pioneers Grant
      In 2025, the Association is launching a new, ambitious grant to support innovative research projects and clinical developments aimed at transforming DMD research and patient care. With funding starting at €500,000 per project, this grant is designed to attract “high risk, high gain” projects.

 

  • Lastly, to further its mission in promoting research on DMD, the association also sponsors and/or organizes conferences, round tables and events aimed at the scientific sector to foster collaboration.

A selection of beneficiaries

Stream 02

The second category is Quality of life and awareness, representing approximately 2% of grantmaking.

  • The first portfolio refers to punctually helping patients and families in dire need of financial support to purchase equipment (i.e. wheelchairs) or pay for essential home adaptations for DMD patients and funding innovative projects with regards to patient mobility and care with the following grant:

 

    • Advancing Patient Mobility (Equipment)
      The Association also occasionally funds projects that aim to improve the quality of life for patients through the development of innovative equipment. This includes supporting research into better wheelchairs and other assistive technologies that enhance mobility and independence for individuals living with DMD and other neuromuscular disorders. We believe that investing in such solutions can have a transformative impact on their day-to-day lives.

 

  • The second portfolio focuses on projects that promote education and raise awareness about DMD through a variety of mediums, including documentaries, books, podcasts, and other forms of media. It also aims to reduce stigma surrounding the condition.

Portraits of researchers

Luis Garcia

Director of Research at CNRS, Director of the ENDICAP laboratory – UMR 1179 Inserm-University of Versailles-St-Quentin, UFR santé Simone Veil, Special scientific advisor for SQY Therapeutics

I’ve had one fixed goal for more than 25 years: find a cure for Duchenne muscular dystrophy. At the time of the last Only Watch we were filing for a clinical trial authorization from the sanitary authorities for a molecule we’ve developed. Now we have that authorization, and we are starting the safety phase. But beware, it’s a sprint type of effort with the duration of a marathon: the trial is starting now but it’s the result of years of work and there are many more steps and developments to come, we must stay focused.

Without Only Watch we would not be where we are today, at a clinical stage, we would still be people with ideas. Thanks to the means it gives us, not only have we shown it’s feasible – we went from ground 0 to clinical stage – but it allows us to have the ambition to develop our technology for other diseases. A big thank you to the Only Watch community.

Watch the interview

Helge Amthor

Professor of pediatrics specialized in muscle diseases in childhood, University Paris-Saclay UVSQ, UFR Simone Veil – Santé, Research Unit U1179, Raymond Poincaré University Hospital

I split my time between consultations at the hospital and research in the laboratory. On the clinical side, I see patients suffering from neuromuscular disorders, amongst whom many with Duchenne muscular dystrophy. Currently I’m also responsible for patients who take part in the clinical trial testing the SQY51 molecule – a clinical trial of which the promoter, SQY Therapeutics, is run by parents of Duchenne patients, and funded by charity funds, from Only Watch.

On the research side, I work with collaborators, some of whom are Master’s or PhD students, on projects aiming to gain a better understanding of the underlying mechanism of DMD, as well as to develop novel therapeutic avenues. This part of my work has also been supported by the charity for years. Being able to pursue both (clinical work and research) has always allowed me to go further in both fields.

Watch the interview

Dr. Angely Mendoza Cardozo

Clinical Chief in the Pediatric Neurology Department at Raymond Poincaré University Hospital, working in the AVANCE 1 clinical trial

This job allows me to develop as a human being and as a neuropediatrician. I thank the Only Project for its support and wish to continue working on this path of hope to improve the life quality of our patients and what we wish for them all, with all our efforts and all our hearts, their healing.

Karim Whabi

MD, PhD, Professor of cardiology at Paris Cité University, head of the reference center for neuromuscular cardiomyopathies in Cochin Hospital, Paris

My key areas of research are the prevention and treatment of heart failure and sudden death, which are the most frequent manifestations of cardiomyopathies in this condition, along with the study of their underlying molecular mechanisms.

I had the opportunity to set up the largest database worldwide dedicated to cardiac disease in Duchenne muscular dystrophy. This project, financed with Only Watch funds, contributed to improving the standard of patient care. I am also involved in various translational research programs involving specialists in both clinical and fundamental research, which are essential to develop innovative treatments to cure this complex genetic disease

Watch the interview

Sadia Belkacem

Member of Pr. Wahbi’s team dedicated to the advancement of knowledge on cardiac damage in Duchenne muscular dystrophy

As a project leader, I coordinate projects led by Prof. Karim Wahbi aimed at improving the cardiac care of DMD patients. These projects are based on the careful collection of quality clinical and genetic data from as many patients as possible in dedicated databases, the analysis of which allows to improve patient care and to set up trials evaluating new therapies.

Christophe Marcelle

Professor, Faculty of Biosciences, Deputy Director, MeLis (CNRS UMR 5284 / INSERM U1314), NeuroMyoGene Institute (INMG), University Claude Bernard Lyon1

As a Professor, I’m extremely keen on supporting the emergence of a next generation of researchers – women and men – and to help them to create bonds with each other, communities, so as to share knowledge, experience and create synergies. Funding granted by the association has allowed me to offer such perspectives to students.
As a researcher myself and a thesis director, it has also allowed me to continue to explore subjects that I would have had to give up otherwise.

Clémence Alibert

PhD student, University Claude Bernard Lyon1

Working under Pr. Marcelle’s guidance on the following thesis project « Muscle fusion as a delivery mechanism to repair ailing muscles from heritable muscle diseases ».

Marisa Jaconi

PhD, Professor at the University of Geneva (UNIGE), Scientific Director of the Lugano Cell Factory, Institute Cardiocentro Ticino‐EOC

Pr. Jaconi‘s ongoing work on « Immortal immunoprivileged myogenic stem cells for gene therapy of muscular dystrophy » spans over two years (2022-2023) and is financed by our charity’s « Paul Pettavino fellowship » granted through the Fondation Suisse pour la Recherche sur les Maladies Musculaires (FSRMM). The goal of her research is to overcome known obstacles to the prospect of effective stem cell therapy for Duchenne muscular dystrophy patients.

Robert M. Kotin

PhD, Adjunct Professor, University of Massachusetts Chan Medical School, Worcester, USA and Chief Science Advisor at Carbon Biosciences

The Only Project’s (AMM) funding was critical for supporting our research leading to one of the widely used production platforms for recombinant adeno-associated virus. The NIH has outlicensed our technology and materials to several companies including uniQure and BioMarin and both have FDA or EMA approve gene therapies based on our platform. (…) It’s unlikely that this would have occurred without the AMM’s support. I’ll always be grateful for the support, confidence, and trust.

Lucie Royer

Molecular biology engineer in Research Unit U1179, University Paris-Saclay, UFR Simone Veil – Santé, Research Unit U1179, working on Duchenne and Becker muscular dystrophy

Watch the interview

A selection of works supported by our association

To give you a better understanding of how we fulfill our mission and the variety of impactful projects we support, we have selected a few recent initiatives funded by the association:

Flagship project: SQY Therapeutics and clinical trial AVANCE 1

AVANCE 1, the first clinical trial using novel therapy SQY51, is currently underway in Garches, France. SQY51 targets mutations that cause Duchenne muscular dystrophy in approximately 10-15% of patients. More specifically, it is an antisense oligonucleotide that works like a “molecular patch” by skipping over the faulty section of the DMD-causing gene, allowing the body to produce a key muscle-preserving protein. The trial, which began in 2023, involves 12 male patients, both children and adults, at various stages of disease progression, and aims to evaluate the safety and efficacy of this promising therapy.

A large-scale natural history study on the heart in DMD

Professor Karim Wahbi is leading a large-scale national history study to better understand how heart muscles are affected in DMD patients. This research, which began in spring 2023 at Necker Hospital, will last at least three years and uses advanced imaging techniques like MRI scans and biomarker analysis to closely monitor how certain heart issues develop and progress over time. The aim is to build the largest collection of cardiac data on DMD patients, expanding upon an already established national registry that the Association previously funded. This expanded database will serve as a vital resource for researchers and clinicians working to improve treatments for heart complications associated with the disease.

Robert Kotin’s study on paroviruses

Dr. Robert M. Kotin’s study, published in November 2022 in PLOS Biology and co-funded by the Bill and Melinda Gates Foundation and the AMM-Only Project entitled “Comparative analysis reveals the long-term coevolutionary history of parvoviruses and vertebrates”. The study explores how parvoviruses, small viruses that can be used in gene therapy, evolved alongside animals over millions of years. His research helps scientists better understand how these viruses can be used safely in treatments like gene therapy, which is important for developing therapies to treat genetic conditions like DMD.

A project on the obstacles to effective stem cell therapy for DMD

Professor Marisa Jaconi’s work on « Immortal immunoprivileged myogenic stem cells for gene therapy of muscular dystrophy » was conducted over two years (2022-2023) and was financed by the Association’s “Paul Pettavino fellowship” granted through our partnership with the Fondation Suisse pour la Recherche sur les Maladies Musculaires (FSRMM). The goal of her research was to overcome known obstacles to the prospect of effective stem cell therapy for Duchenne muscular dystrophy patients.

Clémence Alibert’s PhD thesis on muscle fusion

PhD student Clémence Alibert’s completed her project titled “Muscle fusion as a delivery mechanism to repair ailing muscles from heritable muscle diseases” under the supervision of Professor Christophe Marcelle at Claude Bernard University in Lyon, France.  Her 3-year project (2022-2024) aimed to develop an effective cell and gene therapy for Duchenne muscular dystrophy, testing the hypothesis that genetically modified blood cells, expressing the muscle fusion genes Myomaker and Myomixer, can be used to repair dystrophic muscles.

An AP-HP hospital group study on heart medications for DMD patients

A study titled “Association between prophylactic angiotensin-converting enzyme inhibitors and overall survival in Duchenne muscular dystrophy-analysis of registry data”, published in March 2021 in the European Heart Journal, was conducted by doctors and scientists from the AP-HP hospital group (Cochin, Hôtel-Dieu, Necker-Enfants Malades). The research looked at data from a patient registry to see whether starting heart medications early, specifically ACE inhibitors, could help DMD patients live longer. The findings showed that patients who took these medications before developing major heart issues had better survival rates. This study adds to growing evidence that proactive heart treatment, even before symptoms appear, is essential for managing DMD and improving patient outcomes.

The LIA-BAHN Laboratory’s work in Monaco

The Association has played a pivotal role in supporting the LIA-BAHN laboratory at the Monaco Scientific Center (CSM), which focuses on biotherapies for neuromuscular disabilities. Established in 2013 with significant backing from the Association, the lab has contributed to key advancements in DMD research. Their published work is essential to the DMD ecosystem, helping to drive therapeutic innovations.

Amalia Stantzou postdoctoral work on skeletal muscle pathophysiology

The translational research work of postdoctoral fellow Amalia Stantzou initiated in 2020 on “Skeletal muscle pathophysiology – Differential expression and restoration of dystrophin at critical expression sites in the muscle fiber” at University of Versailles Saint-Quentin-en-Yvelines (France). Her work aims to deepen our understanding of how dystrophin, the essential protein missing in DMD, is differentially expressed in muscle fibers and how it can be restored to its most critical sites to improve muscle function in DMD patients.

A 3D simulator for better diagnosis and treatment

Jean-Paul Carta’s 3D “Phenix” simulator (including a physical platform and VR contents), an innovative technological project that can improve the understanding and treatment of neuromuscular and neurological diseases in a new way thanks to new data capture including sensory stimulation as well as offer recreational multidimensional experiences for wheelchair users.

Applying for a grant

We are eager to support researchers in their endeavors to bring solutions for patients.

Information on applying for grants