[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"dystrophinopathy-symptomatic-female-carrier\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:dystrophinopathy-symptomatic-female-carrier":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,45,68],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":20,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":28,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100640890","duchenne-electronic-health-record-study-100640890",false,"NCT07609394","Duchenne Electronic Health Record Study","Duchenne Outcomes Research Interchange Data Enrichment Through EHR Extraction","Inclusion Criteria:\n\n* Duchenne or Becker muscular dystrophy or female carrier\n* Must be a patient at an institution that has an established EHR integration set up with PPMD's Interchange\n* Must provide consent to have their EHR data pushed to the Interchange and linked to existing Registry data, if applicable\n\nExclusion Criteria:\n\n* Individuals with other forms of muscular dystrophy\n* Individuals who do not provide consent\n\nIndividuals with Duchenne\u002FBecker who have severe mobility\u002Fstrength issues need to provide consent and participate with assistance from a caregiver. Adults with communication impairments and\u002For intellectual disabilities (considered the \"decisionally impaired\" group for purposes of this study) will be able to consent with the assistance of the adults who are designated Legally Authorized Representative (LAR). Without assistance, this group will be excluded from participation because the consent process.","ALL",{"count":18,"type":19},2500,"ESTIMATED","20 Years","OBSERVATIONAL","This study aims to collect retrospective and prospective, long-term data of patients with dystrophinopathy (including Duchenne, Becker, and female carriers) through electronic transfer. At select clinics across the United States, electronic health record (EHR) data from consented patients will be pushed into PPMD's Duchenne Outcomes Research Interchange (the Interchange), where the EHR data can be combined with patient-reported data from The Duchenne Registry. By combining this data in a central hub, we will gain a more complete picture of Duchenne and Becker muscular dystrophy, allowing researchers and clinicians to develop treatments faster and to improve and refine the standards of care for Duchenne and Becker. The ultimate goal is to optimize function, quality of life, and survival of Duchenne and Becker patients.\n\nEHR data collected will be fully identifiable retrospective data for core clinical data elements going back ten years (as available) from the date of consent; going back one year for retrospective clinical notes from the date of consent; and prospectively collecting both core clinical data elements and clinical notes. Information collected will align with the FHIR U.S. core data elements, also known as the Common Clinical Data Set.\n\nPPMD partnered with Prometheus Research (an IQVIA company), an industry leader in health data informatics, to launch both the EHR Study and the Interchange. All data is stored securely and in accordance with strict industry standards and patient privacy laws. Participation in the EHR data extraction is voluntary, and a patient can withdraw consent at any time.",[24,25,26,27],"Duchenne Muscular Dystrophy (DMD)","Becker Muscular Dystrophy","Dystrophinopathy","Dystrophinopathy Symptomatic Female Carrier",[29,30,26,31],"Duchenne","Becker","Muscular Dystrophy","RECRUITING","2026-05-21",{"date":35,"type":36},"2026-05-27","ACTUAL",{"date":38,"type":36},"2022-12-01",{"date":40,"type":19},"2072-12",{"name":42,"class":43},"The Duchenne Registry","OTHER",10,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":42,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":51,"targetDuration":53,"studyType":21,"phases":4,"briefSummary":54,"conditions":55,"keywords":58,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":59,"lastUpdatePostDateStruct":60,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":67},"100214523","the-duchenne-registry-100214523","NCT02069756","The Duchenne Registry: An International, Patient-Report Registry for Individuals With Duchenne and Becker Muscular Dystrophy (Member of TREAT-NMD Neuromuscular Network)","Inclusion Criteria:\n\n* Diagnosis of Duchenne or Becker muscular dystrophy; Manifesting female carriers and asymptomatic female carriers also included in registry.\n\nExclusion Criteria:\n\n* Diagnosis of any other type of muscular dystrophy (including limb-girdle muscular dystrophy).",{"count":52,"type":19},10000,"40 Years","The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of the Registry is to connect Duchenne and Becker patients with actively recruiting clinical trials and research studies, and to educate patients and families about Duchenne and Becker care and research. At the same time, The Duchenne Registry is a valuable resource for clinicians and researchers in academia and industry, allowing access to de-identified datasets provided by patients and their families-information that is vital to advances in the care and treatment of Duchenne. The Duchenne Registry is a member of the TREAT-NMD Neuromuscular Network.",[56,25,26,27,57],"Duchenne Muscular Dystrophy","Dystrophinopathy Female Carrier",[29,30,31],"2026-05-05",{"date":61,"type":36},"2026-05-08",{"date":63,"type":36},"2007-10",{"date":65,"type":19},"2047-10",{"name":42,"class":43},1,{"id":69,"slug":70,"hasResults":11,"nctId":71,"briefTitle":72,"officialTitle":73,"acronym":74,"eligibilityCriteria":75,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":76,"targetDuration":78,"studyType":21,"phases":4,"briefSummary":79,"conditions":80,"keywords":81,"overallStatus":86,"whyStopped":4,"lastUpdateSubmitDate":87,"lastUpdatePostDateStruct":88,"startDateStruct":90,"completionDateStruct":92,"leadSponsor":94,"locationsCount":96},"100623860","registry-for-duchenne-and-becker-muscular-dystrophy-100623860","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",{"count":77,"type":19},1500,"15 Years","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.",[24,25,27],[82,56,25,31,26,83,84,85],"Muscular Dystrophies","FAIR Principles","Neuromuscular Diseases","Health Related Quality of Life (HRQoL)","NOT_YET_RECRUITING","2026-02-06",{"date":89,"type":36},"2026-02-11",{"date":91,"type":19},"2026-04",{"date":93,"type":19},"2041-06",{"name":95,"class":43},"Dr. Andreas Ziegler",2]