View clinical trials related to Muscular Dystrophies.
Filter by:The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies. We will follow participants' experience in this study for 10 years after the end of their previous study. Participants will have 1 annual onsite visit and a few annual remote visits. The exact number of remote visits will be decided by their study doctor.
The abdominal muscles play an important role in stabilizing the trunk and providing postural stability. Children with Duchene muscular dystrophy have weak muscles, which may impair postural adjustments. These postural adjustments are required for gait and dynamic balance during the daily living activities.
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping. Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers. Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.
This study was planned to determine neuropsychological profiles of children with Duchenne Muscular Dystrophy and investigation of its effects on motor functions & compare to typically developed peers.
This study is designed to evaluate the feasibility, wearability and participant satisfaction of novel outcome assessment tools in DMD patients which are performed in the home environment.
The goal of this clinical trial is to evaluate the impact of using the Nomad powered KAFO in people who have had a musculoskeletal or neurological injury that has affected their ability to walk. The main questions it aims to answer are to quantify the effectiveness of the Nomad in improving mobility, balance, frequency of falls, and quality of life in individuals with lower-extremity impairments compared to their own brace, over three months of daily home and community use. Participants will: - Wear a sensor that records everyday activities and mobility. - Perform measures of mobility and different activities of participation using their own brace. - Perform measures of mobility and different activities of participation using the Nomad powered KAFO
Although there are studies showing that the effect on motor performance over time in children with DMD is associated with a decrease in the level of physical activity, no publication has been found that directly examines the relationship between cognitive functions and physical activity level. Therefore, the aim of our study is to investigate the relationship between physical activity level and cognitive functions in children with DMD.
This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
The most common muscular dystrophy among pediatric neuromuscular diseases is Duchenne Muscular Dystrophy (DMD). There is no consensus on a standardized physiotherapy and rehabilitation program or exercise prescription in DMD. Motor imagery (MI) is defined as visualizing motor activities in one's mind without performing any movement. There are studies examining the effectiveness of motor imagery in stroke, cerebral palsy, Parkinson's, peripheral facial paralysis, and phantom pain. This study is aimed to examine the effect of motor imagery on gait and balance functions in children with Duchenne Muscular Dystrophy. Boys residing in Istanbul Turkey, between the ages of 5 and 12, with a diagnosis of DMD who have not lost their ability to ambulate independently will be included in the study. The included individuals will be divided into two groups due to randomization: Group A (Control Group Physiotherapy and Rehabilitation Program) and Group B (Additional Motor Imagery Training to Intervention Group Physiotherapy and Rehabilitation Program). While the physiotherapy and rehabilitation program is applied to the participants in Group A with 40-minute sessions on 2 non-consecutive days of the week for 8 weeks, the participants in Group B will receive an additional 25-30-minute motor imagery program to the physiotherapy and rehabilitation program. Participants were tested with Kinovea Gait Analysis, Timed Up and Go Test, 2 Minute Walking Test, Motor Function Rating Scale for Neuromuscular Diseases, timed performance tests, Pediatric Berg Balance Scale, Pediatric Fear of Fall Questionnaire (Ped-FOF) before and after the program. will be evaluated later. IBM SPSS (Statistical Package for Social Sciences) statistical program version 22.0 will be used for statistical analysis. The conformity of the variables to the normal distribution will be determined by the "Shapiro-Wilk Test". If the variables show normal distribution, the variation within the group will be analyzed with the "Paired Sample T Test", if not, the "Wilcoxon Test" will be analyzed. In the comparison between groups, if the variables show normal distribution, it will be done with the "Independent T Test" in independent groups and the "Mann Whitney U Test" if they do not show normal distribution. Categorical data distributions will be evaluated with the "Chi-square test". In all analyses, p<0.05 will be considered statistically significant.