Active clinical trials for "Muscular Dystrophies"

Duchenne muscular dystrophy (DMD) is a debilitating neuromuscular disease that causes muscle breakdown, weakness, and eventual death. Over the last 40 years parents have received little guidance on the potential of exercise as a therapeutic strategy to maintain muscle function. It is well known that high intensity exercise and eccentric contractions can result in muscle damage in dystrophic muscle, yet the absence of muscle loading will conversely result in muscle wasting. Recent research in rodent models and milder forms of muscular dystrophy supports earlier studies that resistance exercise may have beneficial effects for maintenance of muscle mass in dystrophic muscle. However, careful and systematic investigation into the safety and feasibility of resistance exercise is needed to consider its implementation in boys with DMD. The goal of this project is to assess the safety and feasibility of a home based mild to moderate-intensity strengthening exercise program in boys with Duchenne muscular dystrophy (DMD). Evidence from milder forms of muscular dystrophy and mouse models of DMD suggests that strengthening exercise may be beneficial for these children, but this area has not been adequately explored using human subjects. The results of this study should provide information to assist in the development of scientifically based recommendations concerning optimal exercise parameters for patients with DMD.

On the basis of published data and the investigators' results indicating that oxidative stress may contribute to the peripheral skeletal muscle dysfunction in patients with FSHD, the investigators propose a study to test whether or not an antioxidant supplementation has a therapeutic interest for patients with FSHD. Their results have important implications for the successful implementation of rational antioxidant therapy in FSHD in which cell loss could be linked to oxidative stress.

Investigators investigated that the effects of kinesilogy taping on balance in patients with Duchenne Muscular Dystrophy

In a study from 2003 the investigators showed that adult patients with very low skeletal muscle mass (spinal muscular atrophy (SMA) type II, Duchenne muscular dystrophy, congenital muscular dystrophy) are prone to develop hypoglycemia during prolonged fasting. Since then case reports have described the same phenomenon with hypoglycemia and metabolic crises in children with low skeletal muscle mass provoked by infection, fasting and surgery. Pathophysiological mechanisms of metabolism have never been investigated in adults or children with SMA II. Thus the investigators studied fat and glucose metabolism during prolonged fasting in patients with SMA II and LAMA 2 and compared results to those found in healthy controls.

Shoulder instability due to muscle weakness is a common problem in disorders of the upper extremities. During arm motion, the scapula acts as a dynamic base for the humeral head. To safely move the shoulder with an exoskeleton for the upper extremities a textile orthosis was developed that stabilizes the scapula against the thorax. The support level of the orthosis is continuously manually adjustable. To test the feasibility of our design and to improve the functionality of the textile orthosis, it needs to be investigated how the orthosis acts on people affected by shoulder instability. The investigators seek to explore how people with shoulder instability respond to the orthosis, and how they may benefit from the orthosis function. Therefore, the range of motion of arm elevation will be compared in different conditions: (i) without any support, (ii) with the support of a trained therapist, and (iii) when the device is engaged at the individual's optimal support level. Additionally, pilot tests will be performed to fix different parameters in our study protocol, such as the the optimal orthosis stiffness level and the ideal number of movement repetitions.

The purpose of the study is to assess the safety, tolerability, and effects of L-Arginine on muscles in boys with dystrophinopathy on corticosteroids. Specifically, to see if L-arginine reduces muscle signal abnormalities on MRI done pre and post 30 days of L-arginine administration.

This is an investigation of the efficacy and safety of CRD007 in Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers.

This study investigated the effects of a 2-part dietary consultation on adherence to an anti-inflammatory diet in individuals with neuromuscular disability. The effects on self-efficacy for adhering to the diet as well as neuropathic pain and depression one month post-consult were also determined.

This study is designed to generate additional data on the effect of ataluren for producing dystrophin protein in nonsense mutation nmDMD participants. This study will evaluate dystrophin levels from participants with nmDMD who currently have been receiving ataluren for ≥9 months. The study will have a single visit (Visit 1).

This project is an adaptation trial, testing the efficacy of an evidence-based community wellness program, Enhance Wellness (http://www.projectenhance.org/enhancewellness.aspx), in a sample of middle and older-aged adults living with multiple sclerosis, spinal cord injury, post-polio syndrome and muscular dystrophy.