View clinical trials related to Muscular Dystrophy, Duchenne.
Filter by:The purpose of the study is to explore the biomarker Fast Troponins response to exercise in patients with Becker muscular dystrophy, Limb-girdle muscular dystrophy and McArdle disease
This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.
This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).
Spinal cord injuries and people with Duchenne Muscular Dystrophy or Infant Spinal Muscular Atrophy (ISA) are prone to pain and pressure sores associated with prolonged sitting. For this reason, it is recommended that people with spinal cord injuries release pressure every 15 to 30 minutes and motorized wheelchair users use the electric positioning functions at least 1 minute every hour. The aim is to prevent and/or reduce pain and pressure sores. These devices could help to observe daily the variability of users' pressure maps, their impact on occupational performance, the link with pain and redness and could propose customized adjustments.
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular dystrophy (DMD). Current standard of care (daily prednisone) is associated with adverse side effects. Evidence from DMD mouse models suggest that weekly dosing provides same efficacy without side effects. Appropriate exercise may also benefit but this area has not been adequately explored.
Children with Duchenne Muscular Dystrophy (DMD) have difficulties towards the end of the ambulatory period, especially in activities that require lower extremity proximal muscle strength such as walking, climbing stairs, standing up without sitting. Stair climbing / descending activity is a complex activity that requires joint stability, correct muscle synergy and timing. When the literature is examined; It has been observed that the performance of stair climb up and down activity in individuals with neuromuscular disease has been evaluated with various clinical applications. In recent studies, there are surface electromyography (EMG) studies evaluating various aspects of stair climbing and descending activity. Surface EMG; is a technique for neuromuscular evaluations that is frequently used in both research and clinical applications, noninvasive, and can be used in areas such as neurophysiology, sports science and rehabilitation. Our study was planned to examine the muscle activations in the lower limb muscles involved in climbing up stairs activity in children with DMD and to compare healthy children with children with DMD and children with different levels of DMD. Hypothesis originating from the investigation: H0: There is no difference in the muscle activations measured by surface electromyography (EMG) of the involved lower extremity muscles during climbing up stairs activity between level 1 and level 2-3 children with early DMD. H1: There is a difference in the muscle activations measured by surface electromyography (EMG) of the involved lower extremity muscles during climbing up stairs activity between level 1 and level 2-3 children with early DMD. H2: There is no difference in the muscle activations measured by surface electromyography (EMG) of the involved lower extremity muscles during climbing up stairs activity between children with DMD and healthy children. H3: There is a difference in the muscle activations measured by surface electromyography (EMG) of the involved lower extremity muscles during climbing up stairs activity between children with DMD and healthy children.
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
The purpose of this low interventional study is to collect data on everyday movement in boys with Duchenne muscular dystrophy (DMD) using wearable activity sensors. The activity sensors could provide useful information beyond what is currently collected by functional (movement, strength) assessments in clinic. This information can help with the understanding of the impact of DMD, and perhaps with how possible treatments can affect this impact.
Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.
The purpose of this project is to devise instrumented insoles capable of accurately measuring gait at each footfall, over multiple hours in any environment. To achieve high accuracy, the investigators will develop a new learning-based calibration framework. Features will be tested in controlled lab settings 39 during a single visit in people with SMA (13), DMD (13) and healthy controls (13) and in 15 participants in real-life environments.