FAIR-DMD is a disease-specific digital ecosystem built on the One4 platform for patients with Duchenne and Becker muscular dystrophy in German-speaking countries and their specialized treatment centers.
From harmonization to therapy monitoring and sustainability: seven goals of the study.
02MethodologyStudy design, data collection, One4 App, One4Care and eConsent.
03BoardBoard, use and access committee, data access charter and project transparency.
04CentersParticipating centers and entry point for additional treatment centers.
05ParticipateWho can participate, what patients can expect and how control is preserved.
06FundingNon-profit study, partner contributions, application-related contributions and funding programs.
07TeamBoard leadership, principal investigator and study coordinating center.
08Study accessDirect access to the FAIR-DMD study system for authorized users.
Duchenne muscular dystrophy (DMD) is an X-linked progressive neuromuscular disease affecting about 1 in 5,000 newborn boys. Without disease-modifying therapy, affected individuals typically lose ambulation in early adolescence. Becker muscular dystrophy (BMD) is usually milder but is based on the same genetic mechanism.
In 2024 and 2025, the first disease-modifying therapies were approved: Vamorolone (Agamree) and Givinostat (Duvyzat). Additional substances, including gene addition therapies and antisense oligonucleotides, are in advanced clinical trials.
Systematically capturing effectiveness, safety and tolerability of these therapies in routine care is the central task of the FAIR-DMD study.
Every patient with Duchenne or Becker muscular dystrophy in German-speaking countries benefits from continuous, data-supported and quality-assured care, independent of treatment location. Internationally connected study data accelerates learning and improves long-term care quality.
FAIR-DMD builds a transparent, independent study on FAIR principles: findable, accessible, interoperable and reusable. Academic sponsorship by the GNP ensures independence in data collection, analysis and publication.
The voices of patients are an integral part of the study structure, not an afterthought but part of the architecture.
FAIR-DMD creates the structural foundation to scientifically accompany new disease-modifying therapies in routine care, generate real-world evidence and develop diagnostics, therapy and care structures on an evidence-based basis.
The study is designed so that interventional study arms can be integrated in the future.
FAIR-DMD pursues seven overarching goals that together form the scientific and care-practice foundation of the study.
Harmonization and standardization - build a quality-assured study and harmonized standards for diagnostics, therapy and follow-up care in the D-A-CH region.
Monitoring new therapies - systematically document effectiveness, safety and tolerability of Vamorolone, Givinostat and future innovative therapies as real-world evidence.
Improving quality of care - structured treatment and follow-up plans plus cross-sector networking between clinics, social pediatric centers and office-based physicians.
Independent research instrument - reliable data basis for scientific analyses and clinical studies, academically led and independent.
International collaboration - close alignment with Swiss-Reg-NMD and European partner registries (EURO-NMD) for multinational analyses and international connectivity.
Patient centering - active involvement of patients and relatives, systematic collection of health-related quality of life and patient-reported outcomes and experiences.
Sustainability and integration - long-term continuation of the study beyond the pilot phase and integration as a standard instrument of care research.
FAIR-DMD collects only data that arises in routine care, supplemented by project-specific quality-of-life data.
FAIR-DMD is a prospective, longitudinal, non-interventional observational study. No additional doctor visits, no additional examinations and no interventions.
Patient data is collected fully digitally through the One4 App, free for Android and iOS. Through the app, patients complete questionnaires, communicate with their treatment center if needed and access therapy-specific information.
Clinical data is captured by the treatment center through the One4Care platform and made available to the study, fully integrated into the clinical workflow.
Consent is given digitally through the One4 App. It is presented clearly, can be withdrawn at any time and is traceable through blockchain.
All patient data is processed pseudonymously and complies with GDPR requirements.
The board sets the content and scientific direction of each project. It steers the work of One4 Health gGmbH within the project framework, not the other way around. The board is appointed by the Society for Neuropediatrics (GNP).
The board also acts as the use and access committee (UAC). Anyone who wants to use data submits a formal data-use request through the One4 platform.
The UAC reviews every request and decides independently. The rules are defined in a charter.
The charter is available and will soon be integrated as a download.
Requested and approved data access will be shown transparently here in the future and persisted via blockchain.
Currently, 26 specialized treatment centers in Germany and 5 centers in Austria participate in the FAIR-DMD study.
Switzerland will be connected in the future through data exchange with Swiss-Reg-NMD.
Berlin · DE
Berlin · DE
Bochum · DE
Bonn · DE
Bremen · DE
Dresden · DE
Erlangen · DE
Essen · DE
Freiburg · DE
Gießen · DE
Göttingen · DE
Greifswald · DE
Halle/Saale · DE
Hamburg · DE
Hamburg · DE
Hannover · DE
Heidelberg · DE
Jena · DE
Kassel · DE
Leipzig · DE
München · DE
Münster · DE
Homburg · DE
Stuttgart · DE
Tübingen · DE
Ulm · DE
Wien · AT
Innsbruck · AT
Salzburg · AT
Linz · AT
Graz · AT
Treatment centers in Switzerland run the Swiss-Reg-NMD registry and will be connected to FAIR-DMD through data exchange in the future.
Swiss patients participate through Swiss-Reg-NMD. The planned data exchange will be pseudonymized into the shared analysis ecosystem.
New centers can be integrated at any time if they care for patients with DMD/BMD.
Interested centers can contact us directly. We clarify medical requirements, technical connection and next steps.
Contact usParticipation in FAIR-DMD is voluntary, free of charge and coordinated with your treatment center.
No additional doctor visits, no additional examinations and no interventions. Data arises in the context of your regular care.
Through the One4 App, patients complete quality-of-life questionnaires and stay in contact with their treatment center.
By participating in the study, you automatically contribute to safety monitoring of new therapies, a legal requirement for every approved therapy (PASS/PAES). You will be transparently informed about every concrete data use.
You can withdraw participation at any time without giving reasons, with no effect on your further medical care. Your data will be deleted upon request unless statutory retention duties apply.
Talk to your treatment center. If your center is not yet participating, contact the FAIR-DMD Study Coordinating Center directly.
FAIR-DMD is a non-profit study supported by the Society for Neuropediatrics and operated in trust by One4 Health gGmbH.
One4 Health is non-profit and financed through partnerships with the life-science industry, funding programs and institutional collaborations.
Our projects are created on behalf of the medical expert community, not in the interest of commercial data exploitation.
Partners finance the development and further evolution of the ecosystem. In return, they receive rule-based, transparent access.
Data-providing treatment centers receive compensation for effort. Specific conditions are published continuously.
FAIR-DMD is led by an academic team under the patronage of the Society for Neuropediatrics.
Board leadership
Ludwig Maximilian University Hospital Munich, Dr. von Hauner Children's Hospital, Head of Pediatric Neurology and Medical Director MUC Hauner.
Principal Investigator
University Hospital Essen, Department of Pediatrics I, Senior Physician Pediatric Neurology.
Study Coordinating Center (SCC)
University Hospital Heidelberg, Section of Neuropediatrics and Metabolic Medicine.
