View clinical trials related to Muscular Dystrophy, Duchenne.
Filter by: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.
The aim of this study was to develop a gait assessment instrument for Duchenne Muscular Dystrophy patients (DMD-GAS), and investigate its validity and reliability.The scale was developed considering the expert opinions which included 10 physiotherapists who had experience in the management of patients with DMD over the 2-round Delphi method, and the Content Validity Index (CVI) was calculated. The final version of the DMD-GAS that was agreed upon the experts consisted of 10 items, and each item scored between 0 and 2. The intra-rater reliability was established by the video analysis of children with a 1-month interval and inter-rater reliability was determined by the scores of 3 physiotherapists. The criterion validity was determined by investigating the relationship between the total score of the DMD-GAS and Motor Function Measure (MFM), 6 Minute Walk Test (6MWT), and the data obtained from GAITRite system.
This study was planned to investigate the parental influence on physical activity (PA) level and participation in ambulatory children with Duchenne muscular dystrophy (DMD). For this purpose, 30 children with DMD between the ages of 8-18, who were between Levels 1-4 according to the Brooke Lower Extremity Functional Classification (BLEFC), were included in the study. The demographic information of the participants and their detailed information about the disease were recorded. Parents' PA level was assessed via International Physical Activity Questionnaire-Short Form (IPAQ-SF); Children's PA level was assessed via Physical Activity Questionnaire for Children (PAQ-C) and pedometer, participation was assessed via Pediatric Outcomes Data Collection Instrument (PODCI), and parental influence was assessed via Children's Physical Activity Correlates (CPAC). Additionally, children's PA interest was assessed via Children's Attraction to Physical Activity (CAPA). SPSS 25 program was used in the statistical analysis of the evaluation results. The mean age of the individuals included in the study was found to be 8,70±0,84. Parental influence evaluations, positive and weak-moderate correlations were determined between CPAC Questionnaire "Parental Influence" sub-dimension with PAQ-C (r=0,582), CAPA (0,432) and PODCI (r=0,372) (p<0,05). A positive, moderate correlation was found between the PA levels of mothers obtained from IPAQ-SF and PAQ-C (p<0,01). The results of the study show that the parents, especially the mother who is the primary caregiver, can be an important factor to improve the PA levels, increase their attraction to PA and participation in children with DMD.
The ARCH study is an open-label, single-center, Phase 1b study of EDG-5506 to assess the safety and pharmacokinetics (PK) of EDG-5506 in adults with Becker muscular dystrophy (BMD). EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers.
This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.
The study will evaluate the effect of a therapeutic dose and a supratherapeutic dose of ITF2357 on the QT/QTc interval.
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo (UNIFESP). The recruited individuals were divided into 4 groups, namely: DMD that used deflazacort (DMD-D); DMD that used Prednisone/Prednisolone (DMD-P); DMD Control with no corticoid use (DMD-C) and Controls with typical development (CTD). The protocol was applied during the evaluation that was carried out at outpatient follow-up visits. To assess the functionality of each patient, the Vignos scales were used to characterize the sample and the Motor Function Measure (MFM) for association with HRV indices. All heart rate records were performed using a cardiofrequencymeter (V800, Polar). After placing the brace and monitor, the individuals were placed in the supine position and remained at rest spontaneously breathing for 25 minutes. For HRV analysis, indexes obtained by linear methods, in the domain of time and frequency, and non-linear methods were used.
The aim of this study was to investigate the effects of trunk training on trunk control, upper extremity, and pulmonary function in children with Duchenne muscular dystrophy (DMD). 26 children with DMD aged 5-16 were included in the study. They were divided into two groups (study and control). The study group exercised with the trunk-oriented exercise program and the conventional exercise program under the supervision of a physiotherapist, whereas the control group underwent the conventional exercise program under the supervision of their families at home for 8 weeks. Trunk control, the upper extremity function and respiratory function test were assessed before and after the 8-week exercise program in this study.
HRV is attained using a Polar RS800CX. Then, evaluated through linear, non-linear and chaotic global techniques (CGT). Forty-five male subjects were included in the DMD group and age-matched with forty-five in the healthy Typical Development (TD) control group. They were assessed for twenty minutes at rest sitting, and then five minutes whilst performing the maze task on a computer.
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD).