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Registry for Duchenne and Becker Muscular Dystrophy

Recruitingn/aAges any - anyAllDr. Andreas Ziegler
NCT07402122 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys. The established "standard of care" has improved prognosis; however, a causal therapy is not yet available. In 2024 and 2025, the first disease-modifying therapies were approved. These include Vamorolone (Agamree®) as a corticosteroid replacement with a more favorable side-effect profile for children aged four and older, and Givinostat (Duvyzat®) as a combination therapy with corticosteroids for ambulatory boys aged six and older. In this context, the FAIR-DMD Registry was initiated. The registry is based on the so-called FAIR principles. The acronym FAIR stands for the data principles Findable, Accessible, Interoperable and Reusable. The international FAIR principles are guidelines for the description, storage, and publica

Sign in to get the eligibility rules as simple questions to take to your doctor.

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📋 Eligibility rules (as published)
Inclusion Criteria: * Genetically confirmed diagnosis of dystrophinopathy * Female carriers only if they show clinical symptoms of a dystrophinopathy * Treatment in one of the participating centers * No age restrictions * Ability to understand the patient information and sign the informed consent * Consent capability by the patient themselves and/or by the parents * Agreement to data exchange between the treating physicians, the telemedicine platform, and the registry * Possession of a tablet or a smartphone Exclusion Criteria: * Missing legally valid consent form from the patient and/or legal guardians * Project content is not understandable to the participant and/or legal guardians * Not in possession of a tablet or smartphone
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What it tests

  • Not listed
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Where

30 sites
LKH Universitätsklinikum Graz
Graz, Austria
Tirol Kliniken, Landeskrankenhaus Innsbruck
Innsbruck, Austria
Kepler Universitätsklinkum Linz
Linz, Austria
Landeskrankenhaus Salzburg
Salzburg, Austria
Wiener Gesundheitsverbund, Klinik Favoriten
Vienna, Austria
Charité - Universitätsmedizin Berlin
Berlin, Germany
DRK Kliniken Berlin Westend
Berlin, Germany
Katholisches Klinikum -Universitätsklinikum der Ruhr-Universität Bochum
Bochum, Germany

…and 22 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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