View clinical trials related to Muscular Dystrophy, Duchenne.
Filter by:The aim of this study was to develop a reliable and valid gait classification scale for Duchenne Muscular Dystrophy (QIGS-DMD). The items of the QIGS-DMD were designed based on the literature review considering existing functional classification scales, gait scales, and the opinions of the physiotherapists who were expertized in rehabilitation of patients with DMD. Content validity was determined based on the opinions of a total of ten expert physiotherapists. Videos were recorded during gait of 69 children with DMD and inter- and intra-rater reliability were examined. Criterion validity was determined according to the relationship between QIGS-DMD and Motor Function Measure (MFM) and Vignos Lower Extremity Rating Scale (VLERS).
Motor imagery (MI) can be defined as a dynamic process in which the person is mentally stimulated without performing the given motor movement. Studies of imagery; demonstrated that it alters a person's ability to learn, performance skills, and important cognitive skills (self-efficacy, self-confidence, effort, motivation). In recent years, it has been shown that motor imagery techniques are used for therapeutic purposes as a current neurorehabilitation approach and that imagery can have positive effects on improving motor activity and functions. However, it has been reported that the biggest difficulty in the use of imagery techniques is the inability to determine to what extent the individual can perform mental representation of movements. For this reason, it is thought that it is necessary to evaluate the motor imagery ability first in order to identify the patients who are suitable for motor imagery training. The Kinesthetic and Visual Imagery Questionnaire (KVIQ) is a motor imagery questionnaire developed for individuals with limited mobility for different reasons. The questionnaire assesses both the visual and kinesthetic dimensions of motor imagery. of the KVIQ; It has also been shown in the literature that it is a valid and reliable questionnaire that enables the appropriate evaluation of motor imagery in different neurological disease groups such as Multiple Sclerosis, Parkinson's disease, and stroke. However, the literature When examined, no evidence was found about the motor imagery ability of individuals with Duchenne muscular dystrophy (DMD). It is foreseen that KVIQ will be especially suitable for patients with DMD of different functional levels, since all its items have been developed to be applied to people with limited physical mobility or physically disabled people in a sitting position. Therefore, in this study, it is aimed to investigate the validity and reliability of the Kinesthetic and Visual Imagery Questionnaire for patients with DMD.
The aim of this study is to translate the "North Star Ambulatory Assessment (NSAA)" scale into Turkish and make its cultural adaptation and to demonstrate the reliability and validity of the Turkish version in patients with ambulatory DMD. For the translation into Turkish, validity and reliability of the NSAA, necessary permission was obtained from the developer of the questionnaire, Prof. Dr. Francesco Muntoni, via e-mail. In the study, first of all, the translation and cultural adaptation process will be completed, and then reliability-validity studies will be carried out.
The aim of this study is to examine the prevalence of lower urinary tract symptoms (LUTS) in children with Duchenne Muscular Dystrophy (DMD) and the relationship between functional level, posture, muscle strength, pelvic floor muscle control, participation in activities of daily living, and quality of life that may be associated with these symptoms. Forty-five children with DMD between the ages of 5-18 (Age: 9.00±3.32 years, Weight: 31,10±12,59 kg, Height: 125,87±18,46 cm) and their families were included in the study. LUTS was assessed with Dysfunctional Voiding And Incontinence Scoring System, functional level with Brooke Upper Extremity Functional Classification and Vignos Scale, posture with the New York Posture Assessment Questionnaire, Baseline Bubble Inclinometer (10602, Fabrication Enterprises Inc. New York, USA) and Baseline Digital Inclinometer (12-1057, Fabrication Enterprises Inc, New York, USA), participation in activities of daily living was assessed with the Barthel Index and quality of life was assessed with the Pediatric Quality of Life Inventory 3.0 Neuromuscular Module. Also, using the Hoggan microFET2 (Hoggan Scientific, LLC, Salt Lake City UT, USA) device, hip flexors, quadriceps femoris muscles, shoulder flexors, elbow extensors, elbow flexors, trunk extensors and flexors were evaluated in terms of muscle strength. Evaluations were made once, and the associated factors were compared in the group with and without LUTS, and the relationship between the factors and the severity of LUTS was examined.
Introduction: Progressive muscle weakness, joint contractures and body alignment disorders seen in patients with Duchenne Muscular Dystrophy (DMD) adversely affect the foot structure of the patients. Objective: The aim of this study is to examine the relationship between foot posture, performance and ambulation in patients with DMD. Method: The patient with ambulatory DMD will be included in the study. The foot postures of the patients will be evaluated with the Foot Posture Index. Relationships between the Foot Posture Index and performance tests (6 minute walk test, timed performance tests (10m walking, Gower's, climb/descend 4 stair)) and the North Star Ambulation Evaluation, an ambulation evaluation, will be examined.
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.
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.