View clinical trials related to Muscular Dystrophy, Duchenne.
Filter by:The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home environments. Functional outcomes will include documenting active range of motion and the ability to perform activities of daily living (ADLs) using the standardized Canadian Occupational Performance Measure (COPM) and the Role Evaluation of Activities of Life (REAL) assessments. Secondary objectives are to assess the safety record and report on adverse events (AEs) and parameters related to device usage, including device usage time and the time required to don/doff the device. Secondary objectives also include characterization of user upper limb performance based on etiology.
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Duchenne Muscular Dystrophy(DMD)
The progressive muscle weakness and contractures of the patients adversely affect their gait and balance. It is known that the disorder of the patients' balance and gait affects their functional capacity. The aim of this study is to examine the effects of bicycle ergometer training on gait and balance in children with Duchenne Muscular Dystrophy. Twenty-four children with DMD included in the study will be divided into two groups as home program and home program+bicycle ergometer training with block randomization method. Home program including stretching, respiratory, range of motion, posture and mild resistance exercise with body weight will be asked to apply 3-5 days a week for 12 weeks, aerobic training will be performed 3 days a week for 12 weeks at 60% of their maximum hearth rate with 40 minutes total duration consisting of 5 min warm up and 5 min cool down period. Gait and balance were evaluated with GAITrite and Bertec Balance Check Screener, successively. Assessments will be applied at pre-training and after 12 weeks of training.
To evaluate the positive effects of a home-based physical therapy intervention added to the usual physical therapy programs performed in children with neurological pathologies that induce sensorimotor impairments that affect their quality of life and the importance of the implications of their families in their treatment.
Duchenne Muscular Dystrophy (DMD) is a progressive inherited disease that affects the muscles and causes functional limitations to varying degrees. It is vital to start physiotherapy follow-ups immediately after diagnosis. Patients with DMD are among the most vulnerable groups who have problems in accessing physiotherapy services during the COVID-19 pandemic. The aim of the study was to investigate the effects of the telerehabilitation program developed to protect the physical health of patients with DMD and not to interrupt their follow-up.
A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 120 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker. CANYON is fully enrolled; GRAND CANYON is currently enrolling.
Duchenne muscular dystrophy (DMD) is a X-linked recessive disorder due to a mutation of the dystrophin gene (Xp21). Dystrophin is a sarcolemmal protein of skeletal and cardiac muscle, and its absence causes progressive muscle degeneration and substitution with fat and connective tissue. The progressive muscle degeneration leads to loss of autonomous walking before the age of 15 years and death for cardiac and/or respiratory failure. There are no specific treatment for DMD, and the standard of care is now based on long-term corticosteroid (CS) use. The studies on bone mass in DMD are very few, but they agree in reporting the presence of a reduced bone mass and an increased rate of fractures probably due to long-term steroid therapy and disuse-osteopenia. The aim of this study, involving 20 ambulant DMD boys (age 7-10 years) has been the evaluation of the effects of low-level mechanical vibrations on bone in a group of ambulant DMD children for 1 year, with RDA-adjusted dietary calcium intake and 25OH vitamin D supplementation.
Duchenne Muscular Dystrophy is a genetic disease that causes progressive muscle weakness. There is now substantial evidence that boys with this disease do not demonstrate age-related gains in their cognitive skills. The goals of this study are (i) to use a technology-enabled neurobehavioral assessment called National Institutes of Health Toolbox Cognition Battery (NIHTB-CB) to assess brain development over time; (ii) engage with key-stakeholders to understand how neurodevelopmental problems like attention-deficit hyperactivity, autism spectrum affects individuals (and/or) families, so that we can understand meaningful effects of a potential treatment at an individual level, and (iii) to investigate using brain magnetic resonance imaging (MRI) changes in brain connectivity.
Purpose: This study aimed to evaluate the construct validity and reliability of the Turkish version of the Upper Limb Short Questionnaire (ULSQ) in Duchenne muscular dystrophy (DMD). Materials and methods: A total of 41 children with DMD have participated in the study. Upper and lower extremities functional levels were assessed with Vignos Scale and Brooke Upper Extremity Functional Rating Scale, respectively. The construct validity of the questionnaire was determined using the correlation between the ULSQ and ABILHAND-Kids. The Cronbach alpha value was calculated to determine internal consistency. To determine test-retest reliability, 17 randomly selected children were evaluated seven days after the first evaluation, and the "Intraclass Correlation Coefficient (ICC)" value was calculated.