[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"muscular-dystrophy-duchenne\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:muscular-dystrophy-duchenne":29},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,58,119,171,196,226,250],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":18,"phases":4,"briefSummary":19,"conditions":20,"keywords":30,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":50,"lastUpdatePostDateStruct":51,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":54,"locationsCount":57},"100612219","managed-access-program-for-del-zota-in-participants-with-dmd-mutations-amenable-to-exon-44-skipping-100612219",false,"NCT07250737","Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping","Managed Access to Investigational Use of AOC 1044 in Participants With DMD Mutations Amenable to Exon 44 Skipping","Key Inclusion Criteria\n\nRollover Participants\n\n* Completed Study EXPLORE44-OLE Treatment Period (through W102)\n* No significant tolerability issues with AOC 1044\n\nNew (Non-Rollover) Participants\n\n* Permanently residing in the US and have a US primary health care provider\n* Documented dystrophin gene mutation that is amenable to exon 44 skipping\n* Age 2 or older at the time of consent\n* If previously treated with gene therapy for DMD, treatment and associated immunosuppressive regimen was more than 12 months before consent and in the opinion of the prescriber, participant has had an unsatisfactory treatment response\n\nKey Exclusion Criteria\n\nRollover Participants\n\n• Prescence of any new condition or worsening of existing condition that could affect participant's safety or ability to comply with the program requirements\n\nNew (Non-Rollover) Participants\n\n* Recently treated with or on a clinical study for another investigation drug\n* Serious respiratory or cardiac dysfunction, or nearing end of life\n* Screening laboratory parameters do not meet protocol requirements\n* History of multiple drug allergies or to any component of AOC 1044\n* Participants who discontinued early from the treatment period of EXPLORE44 or EXPLORE44-OLE","MALE","2 Years","EXPANDED_ACCESS","The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.",[21,22,23,24,25,26,27,28,29],"Muscular Disorders, Atrophic","Muscular Diseases","Musculoskeletal Diseases","Neuromuscular Diseases","Nervous System Diseases","Genetic Diseases, Inborn","Genetic Diseases, X-Linked","Muscular Dystrophies","Muscular Dystrophy, Duchenne",[31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48],"Avidity","Avidity Biosciences","AOC 1044","AOC 1044-CS1","AOC 1044-CS2","EXPLORE44","EXPLORE44-OLE","Del-zota","delpacibart zotadirsen","DMD","exon skipping therapy","dystrophin","managed access","expanded access","pre-approval access","compassionate use","MAP","EAP","AVAILABLE","2026-06-26",{"date":52,"type":53},"2026-06-29","ACTUAL",{"name":55,"class":56},"Avidity Biosciences, Inc.","INDUSTRY",14,{"id":59,"slug":60,"hasResults":11,"nctId":61,"briefTitle":62,"officialTitle":63,"acronym":64,"eligibilityCriteria":65,"healthyVolunteers":11,"sex":16,"minAge":66,"maxAge":67,"enrollmentInfo":68,"targetDuration":4,"studyType":71,"phases":72,"briefSummary":74,"conditions":75,"keywords":86,"overallStatus":109,"whyStopped":4,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":118},"100638348","phase-3-efficacy-safety-and-tolerability-of-zeleciment-rostudirsen-dyne-251-administered-intravenously-every-4-weeks-in-ambulatory-participants-with-duchenne-muscular-dystrophy-forzetto-100638348","NCT07608432","Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)","A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping","FORZETTO","Inclusion Criteria:\n\n* Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping .\n* Rise From Floor (RFF) time must be \\\u003C 10 seconds for both screening assessments .\n* Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a stable dose during the Placebo-Controlled Period of the study (unless dose adjustment is required by weight change)\n\nExclusion Criteria:\n\n* Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization\n* Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to randomization\n* Any change in prophylaxis\u002Ftreatment for congestive heart failure (CHF) within 12 weeks prior to randomization\n* Receipt of eteplirsen within 1 week prior to randomization\n* Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization\n* Receipt of givinostat within 12 weeks prior to randomization\n* Receipt of gene therapy at any time\n\nNote: Other inclusion or exclusion criteria may apply","4 Years","18 Years",{"count":69,"type":70},90,"ESTIMATED","INTERVENTIONAL",[73],"PHASE3","The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.",[76,29,77,40,28,78,79,80,81,82,83,84,85],"Duchenne Muscular Dystrophy (DMD)","Muscular Dystrophy (DMD)","Muscular Dystrophy in Children","Muscular Dystrophy, Duchenne Type","Muscular Dystrophy, Duchenne and Becker Types","Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)","Genetic Disease, Inborn","Genetic Disease, X-Linked","Congenital, Hereditary, and Neonatal Diseases and Abnormalities","Neuromuscular Diseases (NMD)",[87,40,88,89,90,91,92,93,94,95,64,96,97,98,99,100,101,102,103,104,105,106,107,108],"Ambulatory","Duchenne Muscular Dystrophy","Duchenne","Dyne","Dyne Therapeutics","DYNE-251","Dystrophy","Exon Skipping","Exon 51","Pediatric","PMO","Muscle Function","Muscular Dystropy, Duchenne","Rise From Floor","RFF","RFF Velocity","Rostudirsen","Time to rise","TTR","TTR Velocity","Zeleciment rostudirsen","Z-rostudirsen","RECRUITING","2026-05-20",{"date":112,"type":53},"2026-05-27",{"date":114,"type":70},"2026-06",{"date":116,"type":70},"2032-10",{"name":91,"class":56},1,{"id":120,"slug":121,"hasResults":11,"nctId":122,"briefTitle":123,"officialTitle":124,"acronym":125,"eligibilityCriteria":126,"healthyVolunteers":11,"sex":127,"minAge":67,"maxAge":4,"enrollmentInfo":128,"targetDuration":4,"studyType":130,"phases":4,"briefSummary":131,"conditions":132,"keywords":147,"overallStatus":109,"whyStopped":4,"lastUpdateSubmitDate":161,"lastUpdatePostDateStruct":162,"startDateStruct":164,"completionDateStruct":166,"leadSponsor":168,"locationsCount":118},"100527411","development-of-non-invasive-prenatal-diagnosis-for-single-gene-disorders-100527411","NCT06147414","Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders","Evaluation of the Diagnostic Performance of Non-Invasive Prenatal Diagnosis for Single Gene Disorders","DANNIgene","Inclusion Criteria:\n\n* pregnant woman with 9 weeks of amenorrhea or more\n* singleton pregnancy\n* undergoing invasive PND in a context of family history of SGD involving the following genes : HBB, CFTR, FMR1, SMN1, DMPK, DMD, NF1, HTT, F8, F9, GCK, L1CAM, PKHD1, or undergoing prenatal counselling in a context of maternal history of diabetes MODY-GCK\n* germinal pathogenic paternal and\u002For maternal mutations previously identified\n* age 18 years old or over\n* signing an informed consent\n\nExclusion Criteria:\n\n* at risk of SGD involving a de novo pathogenic mutation in a previous child\n* woman under legal protection","FEMALE",{"count":129,"type":70},550,"OBSERVATIONAL","Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnosis for single-gene disorders (SGD-NIPD). This can be performed from 9 weeks of amenorrhea and offers an early, safe and accurate definitive diagnosis without the miscarriage risk associated with invasive procedures. One of the major difficulties is distinguishing fetal genotype in the high background of maternal cfDNA, which leads to several technical and analytical challenges. Besides, unlike noninvasive prenatal testing for aneuploidy, NIPD for monogenic diseases represent a smaller market opportunity, and many cases must be provided on a bespoke, patient- or disease-specific basis. As a result, implementation of SGD-NIPD remained sparse, with most testing being delivered in a research setting.\n\nThe present project aims to take advantage of the unique French collaborative network to make SGD-NIPD possible for theoretically any monogenic disorder and any family.",[133,134,135,136,137,138,29,139,140,141,142,143,144,145,146],"Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including","Sickle Cell Disease","Cystic Fibrosis","Fragile X Syndrome","Proximal Spinal Muscular Atrophy","Myotonic Dystrophy","Muscular Dystrophy, Becker","Neurofibromatosis-Noonan Syndrome","Huntington Disease","Hemophilia A","Hemophilia B","MODY2 Diabetes","X-Linked Hydrocephalus","Autosomal Recessive Polycystic Kidney Disease",[148,149,150,151,152,153,154,155,156,157,158,159,160],"Gene HBB","Gene CFTR","Gene FMR1","Gene SMN1","Gene DMPK","Gene DMD","Gene NF1","Gene HTT","Gene F8","Gene F9","Gene GCK","Gene L1CAM","Gene PKHD1","2026-04-20",{"date":163,"type":53},"2026-04-23",{"date":165,"type":53},"2024-10-23",{"date":167,"type":70},"2027-05",{"name":169,"class":170},"Assistance Publique - Hôpitaux de Paris","OTHER",{"id":172,"slug":173,"hasResults":11,"nctId":174,"briefTitle":175,"officialTitle":176,"acronym":177,"eligibilityCriteria":178,"healthyVolunteers":11,"sex":16,"minAge":179,"maxAge":67,"enrollmentInfo":180,"targetDuration":4,"studyType":130,"phases":4,"briefSummary":182,"conditions":183,"keywords":185,"overallStatus":187,"whyStopped":4,"lastUpdateSubmitDate":188,"lastUpdatePostDateStruct":189,"startDateStruct":191,"completionDateStruct":192,"leadSponsor":194,"locationsCount":118},"100632302","a-multicenter-cohort-study-of-duchenne-and-becker-muscular-dystrophy-in-western-chinese-children-100632302","NCT07511920","A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children","A Real-World, Multicenter Cohort Study on the Natural History of Duchenne and Becker Muscular Dystrophy in Children From Western China","WEST-DBMD","Inclusion Criteria:\n\n* Male participants with genetically confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD)\n* Age range: 1 to 18 years old (adjust to your actual age limit)\n* Ability to complete study assessments and follow-up visits\n* Participants or legal guardians provide written informed consent\n\nExclusion Criteria:\n\n* Participants with other neuromuscular disorders that may confound natural history data\n* Participation in another interventional clinical trial that could affect disease progression\n* Severe comorbidities that prevent completion of study assessments\n* Inability to provide informed consent or comply with study procedures","1 Year",{"count":181,"type":70},500,"This is a prospective, multicenter, longitudinal observational cohort study aimed at understanding the progression of Duchenne Muscular Dystrophy (DMD). The primary objective is to identify and integrate key biomarkers from multiple sources-including motor function assessments, body composition (muscle and fat distribution), clinical laboratory tests, and cardiopulmonary imaging-to delineate comprehensive disease trajectories. By analyzing how these factors change over time in a large cohort, the study seeks to develop a robust model that can identify patterns of disease progression. The ultimate goal is to generate evidence that may aid in forecasting individual patient outcomes and inform the future development of personalized rehabilitation and therapeutic strategies.",[139,184,77,29],"Muscular Dystrophy",[186],"Duchenne Muscular Dystrophy; Becker Muscular Dystrophy; Natural History; Multicenter Cohort Study; China; Predictive Model","NOT_YET_RECRUITING","2026-03-30",{"date":190,"type":53},"2026-04-06",{"date":161,"type":70},{"date":193,"type":70},"2028-12-31",{"name":195,"class":170},"West China Second University Hospital",{"id":197,"slug":198,"hasResults":11,"nctId":199,"briefTitle":200,"officialTitle":200,"acronym":4,"eligibilityCriteria":201,"healthyVolunteers":11,"sex":202,"minAge":203,"maxAge":4,"enrollmentInfo":204,"targetDuration":4,"studyType":71,"phases":206,"briefSummary":208,"conditions":209,"keywords":4,"overallStatus":109,"whyStopped":4,"lastUpdateSubmitDate":217,"lastUpdatePostDateStruct":218,"startDateStruct":220,"completionDateStruct":222,"leadSponsor":224,"locationsCount":118},"100544013","the-effect-of-a-muscle-mimicking-fabric-type-shoulder-orthosis-on-functional-movements-of-the-upper-limb-in-patients-with-neuromuscular-disorder-100544013","NCT06363357","The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder","Inclusion Criteria\n\n1. Patients with a confirmed diagnosis of a neuromuscular disease (NMD) by genetic testing, muscle biopsy, or electrodiagnostic studies, presenting with prominent upper limb muscle weakness. Examples include:\n\n   1. Muscular Dystrophies: Duchenne\u002FBecker Muscular Dystrophy (DMD\u002FBMD), Limb-Girdle Muscular Dystrophy (LGMD), Facioscapulohumeral Muscular Dystrophy (FSHD), etc.\n   2. Motor Neuron Diseases: Spinal Muscular Atrophy (SMA, Types 2 and 3), Amyotrophic Lateral Sclerosis (ALS, upper limb-dominant), etc.\n   3. Peripheral Neuropathies: Charcot-Marie-Tooth (CMT) disease, etc.\n   4. Other Neuromuscular Conditions: Including but not limited to cervical spinal cord injury.\n2. Aged over 10 years.\n3. A score of 2 to 5 on the Brooke Upper Extremity Functional Rating Scale.\n4. Manual Muscle Test (MMT) grade of less than 3 for shoulder abduction.\n5. Ability to provide written informed consent from the participant and\u002For their legal representative, indicating willingness to participate in the study.\n\nExclusion Criteria\n\n1. Unwillingness or inability to provide informed consent.\n2. A score of 1 or 6 on the Brooke Upper Extremity Functional Rating Scale.\n3. Cognitive impairment severe enough to interfere with the proper use of a shoulder orthosis.\n4. Any other condition which, in the opinion of the investigator, would make study participation inappropriate or unsafe for the patient.","ALL","10 Years",{"count":205,"type":70},30,[207],"NA","The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder.\n\nThe main questions it aims to answer are:\n\n* What is the impact of the muscle-mimicking, fabric-type shoulder orthosis on upper limb functional movements in patients with neuromuscular disorder?\n* Are there observable differences in upper limb function when the shoulder orthosis is worn versus when it is not?\n\nParticipants will:\n\n* Receive education on how to wear and use the shoulder orthosis.\n* Undergo evaluations, including assessment of upper limb performance, shoulder muscle strength testing, active range of motion measurements, assessment of functional workspace, goal attainment scale evaluation, surface electromyography, physiological measurements such as blood pressure and heart rate, fatigue assessment, and assessment for any musculoskeletal or skin-related issues.\n\nResearchers will compare neuromuscular disorder patients before and while wearing and operating the shoulder orthosis to see if there are any significant effects on variables such as upper limb function, range of motion, functional workspace, goal attainment scale, and surface electromyography.",[29,210,211,85,212,213,214,215,216],"Orthotic Devices","Upper Extremity","Fascioscapulohumeral Muscular Dystrophy","Spinal Muscular Atrophy (SMA)","ALS (Amyotrophic Lateral Sclerosis)","LGMD","SCI - Spinal Cord Injury","2025-11-25",{"date":219,"type":53},"2025-12-03",{"date":221,"type":53},"2024-04-20",{"date":223,"type":70},"2025-12-31",{"name":225,"class":170},"Seoul National University Hospital",{"id":227,"slug":228,"hasResults":11,"nctId":229,"briefTitle":230,"officialTitle":230,"acronym":231,"eligibilityCriteria":232,"healthyVolunteers":11,"sex":202,"minAge":4,"maxAge":4,"enrollmentInfo":233,"targetDuration":4,"studyType":71,"phases":235,"briefSummary":236,"conditions":237,"keywords":4,"overallStatus":109,"whyStopped":4,"lastUpdateSubmitDate":241,"lastUpdatePostDateStruct":242,"startDateStruct":244,"completionDateStruct":246,"leadSponsor":248,"locationsCount":118},"100580145","transcriptomic-analysis-to-put-an-end-to-misdiagnosis-in-patients-with-rare-muscle-diseases-100580145","NCT06833489","Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases","ARNseq-Musc","Inclusion Criteria:\n\n* patients with rare genetic muscle diseases who have benefited from high-throughput sequencing analysis (panel of 200 genes defined by the FILNEMUS Rare Neuromuscular Disease Network) carried out at the Molecular Genetics Laboratory, Medical Genetics Department, Timone Enfant Hospital since 2017.\n\nThis criterion is necessary to limit the analysis to patients with muscular diseases among all the patients analysed by the Molecular Genetics Laboratory.\n\n* this genetic analysis did not identify pathogenic variants explaining the patient's phenotype This criterion is necessary in order to include only patients in diagnostic error.\n* a muscle biopsy of the patient is available in the Biological Resources Centre (CRB) at the AP-HM.\n\nExclusion Criteria:\n\n* Patients with no muscle biopsy available in the CRB.\n* Patients with an established molecular diagnosis.\n* Patients for whom RNA extraction from a muscle biopsy sample did not yield RNA of sufficient quality (INR \\>7) will be excluded from the study. A maximum of two extraction attempts will be performed.",{"count":234,"type":70},50,[207],"Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases. The researchers hypothesise that some of these misdiagnosed patients carry pathogenic RNA (transcript) disrupting variants that were not identified by DNA sequencing. In fact, DNA sequencing analyses can be negative despite the presence of a pathogenic variant that disrupts RNA splicing or expression, causing a genetic disease. For this reason, RNA sequencing can provide a diagnosis in patients who have not been diagnosed by DNA sequencing, thus putting an end to diagnostic wandering. Thus, as a descriptive prevalence study, the objectives are first to determine the rate of positive diagnoses made by the RNAseq approach in patients with muscle diseases that have not yet been diagnosed, and then to identify the genomic characteristics of the pathogenic variants identified in patients by RNAseq analysis, in order to facilitate the identification of this type of variant in future patients.\n\n50 patients will be included in this study during 2 years.",[238,29,139,239,240],"Rare Genetic Muscle Diseases","Congenital Myopathy","Pompe Disease (Infantile-Onset)","2025-02-17",{"date":243,"type":53},"2025-02-18",{"date":245,"type":70},"2025-03-01",{"date":247,"type":70},"2027-03-01",{"name":249,"class":170},"Assistance Publique Hopitaux De Marseille",{"id":251,"slug":252,"hasResults":11,"nctId":253,"briefTitle":254,"officialTitle":255,"acronym":256,"eligibilityCriteria":257,"healthyVolunteers":11,"sex":16,"minAge":258,"maxAge":259,"enrollmentInfo":260,"targetDuration":4,"studyType":71,"phases":262,"briefSummary":263,"conditions":264,"keywords":4,"overallStatus":109,"whyStopped":4,"lastUpdateSubmitDate":265,"lastUpdatePostDateStruct":266,"startDateStruct":268,"completionDateStruct":270,"leadSponsor":272,"locationsCount":118},"100444377","phase-3-the-efficacy-and-safety-of-metoprolol-as-add-on-treatment-to-standard-of-care-in-preventing-cardiomyopathy-in-patients-with-dmd-100444377","NCT05066633","The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD","The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With Duchenne Muscular Dystrophy Aged 8-16 Years. A Randomized, Double-blind, Placebo-controlled Study","MeDMD","Inclusion Criteria:\n\n* Subject's parent(s) or legal guardian(s) has (have) provided written informed consent, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to requirements (\\>16 years old)\n* Stated willingness to comply with all study procedures and availability for the duration of the study\n* Ability to take oral medication and be willing to adhere to the study intervention regimen\n* Subject has confirmed diagnosis of DMD, as defined as clinical picture consistent with typical DMD and: i) Dystrophin immunofluorescence and\u002For immunoblot showing complete dystrophin deficiency, or ii) Identifiable mutation within the DMD gene (deletion\u002Fduplication of one or more exons), where reading frame can be predicted as 'out-of-frame' or, iii) Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e., nonsense mutation, deletion\u002Fduplication leading to a downstream stop codon)\n* Taking ACEi treatment at minimum required doses for at least 30 days\n\nExclusion Criteria:\n\n* Current or previous permanent use of any beta-blocker medication\n* Treatment with another investigational drug or other intervention within 3 months prior to screening\n* Clinically significant bradycardia at rest or by Holter ECG, based on age and sex adjusted normal values, atrioventricular block higher than first degree at rest, or second degree Wenckebach at night, pauses longer than 2.5 seconds\n* Presence of pacemaker or ICD\n* Clinical signs or symptoms of heart failure\n* Left ventricular Ejection Fraction (LVEF) \\\u003C57% (assessed by Teichholtz echocardiography)\n* Inability to obtain adequate quality echocardiography images (necessary to monitor for primary endpoint and safety)\n* Known allergic reactions to components of the IMPs","8 Years","17 Years",{"count":261,"type":70},150,[73],"The study includes 150 patients with DMD diagnosis confirmed by genetic testing, 8-16 years old (≥8 and \\\u003C17) at the study entry with a follow-up of up to 5 years. Random enrollment of a patient to one of two groups (intervention or control) takes place after pre-screening and screening stage starts the first phase of the trial. To be eligible for participation in the study, patients must receive standard of care cardiac therapy, which is an Angiotensin-converting-enzyme inhibitor (ACEi) for at least one-month prior to enrollment. A major part of the trial is equal for all patients - who will be receiving indistinguishable investigational medicinal products (IMPs), the drug metoprolol succinate or placebo. As a part of the clinical trial, diagnostic examinations evaluating progression of the disease, will be performed periodically. In addition, all patients will be monitored at home. Heart rate, blood pressure and patients' personal well-being will be controlled using telemedicine technologies. Additional visits in the research center will be provided if any adverse events occur. This model will be continued for 30 months from the enrollment of a first patient. After this period the first drug efficiency analysis will be performed. After that, the intervention may be continued or in case of negative impact of the intervention on patients' health and well-being, terminated with further patients monitoring.",[29],"2021-09-23",{"date":267,"type":53},"2021-10-04",{"date":269,"type":53},"2021-08-18",{"date":271,"type":70},"2026-06-30",{"name":273,"class":170},"Medical University of Gdansk"]