View clinical trials related to Duchenne Muscular Dystrophy.
Filter by: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.
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 related CM. Despite risk factors for hyperglycemia, including the use of glucocorticoids, low muscle mass, 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. In this Aim of the study, the investigators will assess the utility of remote wearable technology to predict changes in traditional metrics of metabolism and cardiac function. In this pilot study, 10 individuals with DMD will undergo cardiac magnetic resonance imaging (CMR) and oral glucose tolerance tests (OGTTs) at baseline and two years. The investigators will remotely assess glycemia (using continuous glucose monitors), HRV (using extended Holter monitors), and activity (using accelerometers) every 6 months over the 2 years and evaluate if changes in wearable metrics predict changes in CMR and OGTT.
The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. The study consists of 3 periods: A Screening Period (up to 45 days), a Treatment and Observation Period (16 weeks), and an Extension Period (108 weeks).
The purpose of this non-interventional study is to evaluate the feasibility of remotely administering the North Star Ambulatory Assessment (NSAA) to participants with Duchenne muscular dystrophy (DMD). The iTakeControl (iTC) software platform will be utilized to remotely administer and score the NSAAs.
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD mutation amenable to exon 50 skipping.
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.
The objective of ActiLiège Next Study is to collect natural history data in Duchenne muscular dystrophy (DMD) (ambulatory and non-ambulatory patients), and in affected patients with Facio-Scapulo-Humeral dystrophy (FSHD), a disease in which results are currently lacking. It consists of obtaining longitudinal data for the patients, as well as normative data for the control subjects, with a particular emphasis on pediatric subjects using the device on both ankles (for walking) in order to qualify the ascent rate of staircase.
Muscular dystrophy (MD) is a group of muscle diseases that results in increasing weakening and breakdown of skeletal muscles over time. The disorders differ in which muscles are primarily affected, the degree of weakness, how fast they worsen, and when symptoms begin. Many people will eventually become unable to walk. Some types are also associated with problems in other organs. The muscular dystrophy group contains thirty different genetic disorders that are usually classified into nine main categories or types. The signs and symptoms consistent with muscular dystrophy are: progressive muscular wasting, poor balance, scoliosis (curvature of the spine and the back), progressive inability to walk, waddling gait, Calf deformation, Limited range of movement, respiratory difficulty, cardiomyopathy and muscle spasms This study aimed to assess the efficacy of Kinesiology Taping on head and trunk control in patients with Duchenne muscular dystrophy
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study. Pre-infusion baseline will be defined as the timepoint just prior to infusion of delandistrogene moxeparvovec from a previous clinical study. Each participant will be followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a previous clinical study. The duration of participation in this study is dependent on the length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec from a previous clinical study.