View clinical trials related to Duchenne Muscular Dystrophy.
Filter by:AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.
This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.
This study aimed to investigate the validity and reliability of 6PBRT in individuals with DMD and its applicability on these patients.
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single IV infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study, dosed sequentially. Cohort 1 will include participants 4 to <6 years of age, inclusive. Cohort 2 will only be opened after dosing and monitoring a subset of participants in Cohort 1. Cohort 2 will include participants 6 to <8 years of age, inclusive. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
Duchenne muscular dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death. Heart failure is a leading cause of death in DMD, but substantial knowledge gaps exist regarding predisposing risk factors. In the general population, hyperglycemia, insulin resistance, and decreased heart rate variability (HRV; reflecting autonomic dysfunction) are associated with cardiomyopathy (CM). It is unclear whether these factors are associated with DMD-CM. Closing this knowledge gap may lead to novel screening and therapeutic strategies to delay progression of DMD-CM, now the leading cause of death in patients with DMD. Despite risk factors for hyperglycemia, including the use of glucocorticoids (GCs), sarcopenia, obesity, and reduced ambulation, little is known regarding glucose abnormalities in DMD. Some of these same risk factors, along with the distance needed to travel for specialty care, present significant barriers to research participation and clinical care for individuals with DMD. Remote wearable technology may improve research participation in this vulnerable population. Therefore, this study will leverage remote wearable technologies to overcome these barriers and define the relationship between dysglycemia and DMD-CM. The goal of this remote study is to evaluate rates of hyperglycemia in individuals with DMD compared to control participants using continuous glucose monitors, and to determine the relationship between hyperglycemia and heart rate variability. Participants will utilize continuous glucose monitors, cardiac monitors, and activity monitors to evaluate glucose levels, heart rate, activity, and sleep.
This study is a single-center, single-arm, non-randomized, open-label, non-controlled, dose-escalation, prospective clinical trial designed to assess the safety, tolerability, and preliminary efficacy of JWK007 injection in pediatric patients with Duchenne Muscular Dystrophy (DMD).
When the field of neurorehabilitation is examined, most of the current physiotherapy and rehabilitation approaches are based on real movements to stimulate damaged motor neural connections through neuroplasticity. However, since studies have shown that similar brain regions are activated during real movement with motor imagery, which is defined as imagining movement without actually revealing the movement, the findings of these studies suggest that motor functions can be improved through neuroplasticity, just like real movement. When the literature especially in the pediatric population is examined; The effectiveness of motor imagery training with children with cerebral palsy was examined and positive results were found. However, there are no such studies on children with DMD. In addition, telerehabilitation-based motor imagery training is a very rare treatment modality that requires further research. Therefore, the aim of the study is to investigate the effect of telerehabilitation-based motor imagery training on motor imagery ability, motor function and physical performance in children with DMD. The secondary aim of the study is to investigate the effects of telerehabilitation-based motor imagery training on psychosocial factors including fatigue and quality of life in children with DMD.
Duchenne Muscular Dystrophy (DMD) is a progressive genetic neuromuscular disease characterized by progressive loss of motor function, respiratory failure, and cardiomyopathy required regular physiotherapy. With the outbreak of the pandemic rehabilitation centers that make up the weekly physiotherapy routine of children with disabilities have slowed down or even stopped their activities. So DMD who have additional diseases such as respiratory muscle weakness, spinal deformity, obesity, and cardiac dysfunction have also been negatively affected. The 'telerehabilitation' method, which is well planned and prepared for the abilities and needs of patients and caregivers, is seen as a good option at this point. Studies, reporting the feasibility and safety of telerehabilitation in joint replacement, multiple sclerosis, and post-operative conditions, report that the length of stay was reduced, there was access to the same level of service regardless of the distance, and there was no travel cost. Despite these advantages, the framework and applicability of telerehabilitation programs have been investigated limited and not focused on effectiveness of telerehabilitation in patients with DMD. According to the current knowledge, telerehabilitation in DMD is a subject that needs to be investigated in terms of its benefits. So, in this study, it was aimed to show the telerehabilitation's feasibility and its effects on performance level, endurance, fall frequency, pulmonary functions, and satisfaction level with the program in individuals with DMD.
The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.