View clinical trials related to Duchenne Muscular Dystrophy.
Filter by:The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy (DMD) age ≥ 8 to < 16 years old receiving corticosteroid therapy.
The goal of this clinical trial aims to establish if there are meaningful benefits to providing a hydrotherapy service for young people with Duchenne muscular dystrophy (DMD). The main aims are to: 1. to allocate a clinical physiotherapist to a project implementing hydrotherapy in young patients with DMD to establish whether there are meaningful benefits to their daily life. 2. to conduct patient and parent interviews to understand the barriers to completing a hydrotherapy intervention and ensure future research addresses meaningful outcomes for those with DMD.
Having and caring for a child with disabilities brings emotional, social and economic difficulties for many families. Families may experience many physiological and psychological problems due to the stress and anxiety they experience. In addition, it is seen that families with children with disabilities give up their existing roles, reduce their participation in social activities, and reach stagnation in their social lives. Mothers are affected psychologically more than fathers and feel lonely. It is stated that mothers believe that they cannot afford everything in the face of the responsibilities they carry and accordingly, they experience emotional and psychological problems such as stress, anxiety, depression, absent-mindedness, forgetfulness and tantrums. Living with a child with a disability causes family members to experience different emotions as mentioned above; families may frequently experience fear, anxiety, guilt, anger and depression. It is reported that mothers of children with DMD experience depression, anxiety about the future and uncertainty more than mothers of healthy children. Families of children with DMD reported that they felt tired and fatigued during the process of caring for the child and had difficulties in participating in social activities and allocating time for themselves. Most of these families stated that they needed psychological and social support. Therefore, it is important to address the psychiatric aspects of families with children with DMD during the disease process. Parental health contributes positively to the health and adaptation of the family in general. Examining the psychiatric symptoms caused by the problems experienced by families related to DMD and how they cope with this stress will be useful in evaluating and addressing these families. In addition, the social support that families with children with disabilities receive from their immediate environment and institutions is also an important issue. It has been reported that social support from relatives, friends, neighbors, organizations and communities increases the psychological resilience levels of families, they feel that they are not alone in the face of problems, and their anxiety levels decrease. In the literature, it is generally mentioned that when the culture of pediatric care is supportive and family-oriented, the care of the patient will undergo a change when transitioning from pediatric care to the adult period. However, studies evaluating the problems experienced by families in the care of patients with DMD, psychiatric symptoms, ways of coping with stress and perceived social support are insufficient. It is important to evaluate the problems experienced by parents in the families of children with DMD in developing skills to cope with the disease process and disease-related problems, and then to provide training in these areas. Because if parents, who are in the role of caregivers, are equipped with knowledge and skills in this context, they will provide better care and be more useful to their children with DMD. In line with this information, the aim of this study was to evaluate the problems experienced by parents of children with DMD, psychiatric symptoms, coping skills with stress and the level of social support they perceive and to implement a psychosocial support-based psychoeducation program related to these areas.
This research aims to improve the quality of life, occupational performance, occupational satisfaction and emotional health of young people with Duchenne muscular dystrophy compared to the classical occupational therapy program. The findings are planned to shed light on the development of new and effective strategies in the rehabilitation of adolescents with Duchenne muscular dystrophy.
This study aims to examine the relationship between upper extremity muscle strength, balance and functional skills of children with DMD.
The aims of the study are to prospectively collect information on several aspects of function in non-ambulant DMD patients by using a structured battery of tests including motor, respiratory and cardiac function
The purpose of this study is to analyze the effectiveness of a 5-weeks respiratory digital intervention program in patients with Duchenne muscular dystrophy and Becker muscular dystrophy.
A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Duchenne Muscular Dystrophy
This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to participants who were randomized to the Usual Care treatment group of the HOPE-Duchenne study (NCT02485938) and completed 12 months of follow-up. The trial will assess the safety and efficacy of two intravenous administrations of CAP-1002, each separated by three months.
Thanks to tele-assessment methods, it may be possible to evaluate DMD patients without traveling to clinical centers. In recent years, the applicability of remote assessment methods in DMD patients, as in many populations, is being investigated. However, studies have generally focused on a single evaluation parameter such as physical function, a special evaluation method or a special evaluation tool. The aim of this study is to investigate whether remote assessment of functional performance and quality of life in DMD patients is valid and reliable. If a valid and reliable tele-evaluation method that includes functional performance and quality of life parameters is found to be valid and reliable, the travel burden on patients and caregivers can be eased, patients' stress and anxiety related to travel can be reduced, caregivers can save time and energy and provide patients with the best possible treatment.