[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100623860":3},{"organization":4,"armGroups":7,"interventions":7,"overallOfficials":8,"centralContacts":17,"locations":25,"responsibleParty":55,"collaborators":7,"id":59,"slug":60,"hasResults":61,"nctId":62,"briefTitle":63,"officialTitle":64,"acronym":65,"eligibilityCriteria":66,"healthyVolunteers":61,"sex":67,"minAge":7,"maxAge":7,"enrollmentInfo":68,"targetDuration":71,"studyType":72,"phases":7,"briefSummary":73,"conditions":74,"keywords":78,"overallStatus":86,"whyStopped":7,"lastUpdateSubmitDate":87,"lastUpdatePostDateStruct":88,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":96},{"fullName":5,"class":6},"University Hospital Heidelberg","OTHER",null,[9,13],{"name":10,"affiliation":11,"role":12},"Ulrike Schara-Schmidt, Prof. Dr.","Universitätsklinikum Essen, Klinik für Kinderheilkunde","PRINCIPAL_INVESTIGATOR",{"name":14,"affiliation":15,"role":16},"Andreas Ziegler, Dr.","Universitätsklinikum Heidelberg, Zentrum für Kinder- und Jugendmedizin","STUDY_DIRECTOR",[18,22],{"name":14,"role":19,"phone":20,"phoneExt":7,"email":21},"CONTACT","004962215632207","FAIR-DMD@med.uni-heidelberg.de",{"name":10,"role":19,"phone":23,"phoneExt":7,"email":24},"0049 201 723 2508","Ulrike.Schara-Schmidt@uk-essen.de",[26,42],{"facility":27,"status":7,"city":28,"state":7,"zip":7,"country":29,"countryCode":30,"cosmosGeoPoint":31,"geoPoint":36,"contacts":37},"Universitätsklinikum Essen Klinik für Kinderheilkunde I","Essen","Germany","DE",{"type":32,"coordinates":33},"Point",[34,35],7.01228,51.45657,{"lat":35,"lon":34},[38,40,41],{"name":39,"role":19,"phone":23,"phoneExt":7,"email":24},"Ulrike Schara-Schmidt, Prof. Dr. med.",{"name":7,"role":19,"phone":7,"phoneExt":7,"email":24},{"name":39,"role":12,"phone":7,"phoneExt":7,"email":7},{"facility":43,"status":7,"city":44,"state":7,"zip":7,"country":29,"countryCode":30,"cosmosGeoPoint":45,"geoPoint":49,"contacts":50},"Universitätsklinik Heidelberg, Zentrum für Kinder- und Jugendmedizin","Heidelberg",{"type":32,"coordinates":46},[47,48],8.69079,49.40768,{"lat":48,"lon":47},[51,54],{"name":52,"role":19,"phone":20,"phoneExt":7,"email":53},"Andreas Ziegler, Dr. med.","Andreas.Ziegler@med.uni-heidelberg.de",{"name":52,"role":12,"phone":7,"phoneExt":7,"email":7},{"type":56,"investigatorFullName":57,"investigatorTitle":58,"investigatorAffiliation":5,"oldNameTitle":7,"oldOrganization":7},"SPONSOR_INVESTIGATOR","Dr. Andreas Ziegler","Head of paedKliPS (Pediatric Clinical Pharmacology Study Center)","100623860","registry-for-duchenne-and-becker-muscular-dystrophy-100623860",false,"NCT07402122","Registry for Duchenne and Becker Muscular Dystrophy","Development and Implementation of a Transparent Registry Based on FAIR Principles for Duchenne and Becker Muscular Dystrophy (DBMD) Under the Auspices of the Society for Neuropediatrics (GNP) - FAIR-DMD","FAIR-DMD","Inclusion Criteria:\n\n* Genetically confirmed diagnosis of dystrophinopathy\n* Female carriers only if they show clinical symptoms of a dystrophinopathy\n* Treatment in one of the participating centers\n* No age restrictions\n* Ability to understand the patient information and sign the informed consent\n* Consent capability by the patient themselves and\u002For by the parents\n* Agreement to data exchange between the treating physicians, the telemedicine platform, and the registry\n* Possession of a tablet or a smartphone\n\nExclusion Criteria:\n\n* Missing legally valid consent form from the patient and\u002For legal guardians\n* Project content is not understandable to the participant and\u002For legal guardians\n* Not in possession of a tablet or smartphone","ALL",{"count":69,"type":70},1500,"ESTIMATED","15 Years","OBSERVATIONAL","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 publication of scientific or administrative data. The FAIR-DMD registry is a disease-specific, academically managed registry for patients with Duchenne and Becker muscular dystrophy (DMD\u002FBMD). Its goal is to systematically collect clinical data, scientifically monitor new disease-modifying therapies in routine care, and create an evidence-based foundation for the further development of diagnostics, therapy, and care structures. Furthermore, the registry collects data on patients' health related quality of live using an app for data entry. The FAIR-DMD Registry is being established under the auspices of the Society for Neuropediatrics (GNP) and operated in close coordination with Swiss Registry for Neuromuscular Disorders (Swiss-Reg-NMD). The GNP is a non-profit professional society that covers the entire spectrum of neuropediatric topics in clinical and cross-sector care. In the planned pilot phase, the GNP will act as trustee for financing. This model creates the opportunity to structurally address central challenges in health services research and establish a high-quality, internationally compatible registry structure. In the long term, the FAIR-DMD Registry aims to significantly improve care for DMD and BMD patients in German-speaking countries, evaluate the effectiveness of new therapies in clinical practice, and establish binding frameworks for quality-assured care.",[75,76,77],"Duchenne Muscular Dystrophy (DMD)","Becker Muscular Dystrophy","Dystrophinopathy Symptomatic Female Carrier",[79,80,76,81,82,83,84,85],"Muscular Dystrophies","Duchenne Muscular Dystrophy","Muscular Dystrophy","Dystrophinopathy","FAIR Principles","Neuromuscular Diseases","Health Related Quality of Life (HRQoL)","NOT_YET_RECRUITING","2026-02-06",{"date":89,"type":90},"2026-02-11","ACTUAL",{"date":92,"type":70},"2026-04",{"date":94,"type":70},"2041-06",{"name":57,"class":6},2]