Active clinical trials for "Muscular Diseases"

Bullying is an epidemic in Canada, and rates may be underreported. Youth with a disability were more likely to be bullied that those without disabilities, specifically if the disability was visible. Research has been conducted on the prevalence and effects of bullying in youth with disabilities such as cerebral palsy, obesity, and chronic pain; however, there is a paucity of research involving youth with muscular dystrophy and congenital myopathies. The objectives of this study are to: (1) measure bullying frequency, (2) describe the types of bullying experiences; and (3) explore barriers and facilitators to dealing with bullying by youth with muscular dystrophy or congenital myopathies and their parents. The objectives will be met by an online survey and qualitative interviews of youth with muscular dystrophy and congenital myopathy and their parents.

To identify determinants of 'good and bad recovery' from ICUAW and build knowledge around the timing of these processes. Also, the investigators want to discover the effects of ICUAW on physical function and HRQoL following aortic surgery.

To assess changes in impedance parameters in Idiopathic Inflammatory Myopathies (IIMs). To assess whether EIM parameters are reflective of disease severity, based on clinical outcome measures of IIMs.

This study shows an evaluation of the correlation between the muscle strength levels and functional autonomy of elderly women.

Background: - Duchenne muscular dystrophy (DMD) is a disease in which the muscles are unable to make the protein dystrophin. Without this protein, the muscles become gradually weaker. A new medicine called GSK2402968 is being tested to see if it can help prevent or slow down this loss of muscle strength. In this study, boys with DMD and healthy volunteers will have different types of imaging studies to see which ones provide the best images of the muscles. This information will help researchers use these imaging techniques to test the safety and effectiveness of GSK2402968 and other agents. Objectives: - To test magnetic resonance imaging and ultrasound techniques that can detect changes in muscles of boys with DMD. Eligibility: Boys who have DMD and are in the GSK2402968 drug test study. Healthy boys of the same age as the above study participants. Design: Participants will be screened with a medical history and physical exam. Healthy volunteers will have one 2-hour visit with three tests. Magnetic resonance imaging (MRI) scans of the skeletal muscles and heart and diaphragm muscles will be carried out. Muscle ultrasound imaging of leg and arm muscles will also be done. Participants should not perform heavy physical activity like school sports or long walks during the week before the visit. Participants in the GSK2402968 study will have the same series of tests as the healthy volunteers. The tests will be given during the study screening phase. They will be repeated after 3 months and 6 months of receiving the study agent (GSK2402968 or placebo) and at 6 months after stopping the GSK study.

Physical training may improve physical capacity and health parameters in various systemic autoimmune diseases, including idiopathic inflammatory myopathies. Therefore, the present study will assess the role of an exercise training program in patients with idiopathic inflammatory myopathies.

Impairment in physical function is a significant problem for survivors of critical illness. There is a growing urgency to develop a core set of outcome measures, which can be adopted in clinical and research practice to evaluate efficacy in response to interventions such as rehabilitation. Phase 1: Development of a new outcome measure. This study aims to examine the development of a single outcome measure which may be able to be utilised across the continuum of recovery of critical illness in the evaluation of physical function. The study will involve examination of two common physical function measures - the Physical Function in intensive care test scored (PFIT-s) and De Morton Mobility Index (DEMMI) and the development of a new measure based on rasch principles which may be able to capture physical functioning changes in individuals with critical illness. Aims: (1) To determine the clinical utility of two physical function measures (DEMMI and PFIT-s) when used in isolation across the hospital admission; and (2) To transform the (15-item) DEMMI and (4-item) PFIT-s into a single measure to evaluate function in intensive care survivors using Rasch analytical principles. Phase 2: Measurement properties of the PACIFIC physical function outcome measure in an independent validation sample.

The primary aim of the project is to apply the non-invasive stimulated muscle force assessment system to i) intensive care unit patients and ii) patients with primary myopathy or subclinic myopathy (e.g., Duchenne muscular dystrophy, Myotonia, MH). The investigators will use this new methodology of force assessment to phenotype patients weakness during their illnesses (depict typical force pattern) and monitor recovery periods, or disease progression, respectively. This new system will be evaluated until the year 2018. The investigators will determine variables of isometric skeletal muscle force. These systems should serve as force monitoring tools and help to guide therapies.

A major contributor to frailty and immobility in the elderly is the age related loss of muscle mass (sarcopenia). Cardiovascular disease (CVD) is the leading cause of mortality in the elderly, with high blood cholesterol and lipids being the major modifiable risk factor. Statins reduce blood cholesterol, but muscle related pain, tenderness and discomfort (myopathy) is an adverse event associated with statin therapy, with older people being at a much greater risk. Statin myopathy presents as muscle aches and weakness, with or without evidence of muscle damage; however the underlying mechanisms responsible for these symptoms are poorly understood. Using an animal model, the applicants have shown the main pathway regulating muscle protein synthesis is inhibited in statin myopathy, and genes regulating muscle protein breakdown, the inhibition of muscle carbohydrate use and inflammation are dramatically increased. Therefore we wish to determine whether these changes are seen in the muscle of older people with symptoms of statin myopathy, and whether this is associated with lower muscle mass and impaired muscle function compared with older people with no history of statin use. Identification of the mechanisms involved in statin myopathy could lead to effective therapy for older people unable to tolerate statins.

The purpose of this study is to develop simple diagnostic screeningtests and investigate potential biomarkers for identifying patients with abnormalities of mitochondrial function, which also can be used as outcome measures in future clinical trials. The study will investigate two submaximal tests: a submaximal handgrip test and a walking test. Furthermore investigators will investigate Acyl-carnitine profiles and GDF-15 levels in patients with mitochondrial myopathy.