View clinical trials related to Muscular Dystrophies.
Filter by:The purpose of this research study is to identify and study changes in muscle in people with facioscapulohumeral muscular dystrophy using magnetic resonance imaging and spectroscopy.
The objective is to determine whether nebivolol, a beta-blockade drug, can prevent the development of heart disease in patients with Duchenne muscular dystrophy aged 10 to 15 year-old.
This is an interventional study on Duchenne muscular dystrophy patients who will be receiving sedation for a muscle biopsy as part of another study.
The purpose of this study is to evaluate and optimize advances in radio frequency (RF) coil magnetic resonance imaging (MRI) technology at Cincinnati Children's Hospital Medical Center (CCHMC).
Background: - Muscular dystrophy can affect the muscles used for heart function and breathing. Treatment usually involves drugs that help improve heart function. However, better types of heart imaging studies are needed to improve treatment of heart problems related to muscular dystrophy. Better heart imaging methods are especially needed for children with muscular dystrophy. Researchers want to test different heart imaging methods in children with muscular dystrophy. They will look at cardiac magnetic resonance imaging (MRI) and standard heart function tests. Objectives: - To develop and test new methods for imaging the heart in children with muscular dystrophy. Eligibility: - Children and adolescents between 8 and 17 years of age who have muscular dystrophy. Design: - Participants will be screened with a physical exam and medical history. - Participants will provide a blood sample at the start of the study. They will also have heart function tests before having the imaging study. - Participants will have a cardiac MRI scan that will last up to 60 minutes. Some tests will require a MRI contrast agent (a drug that helps the image appear more clearly on the scan).
The hypotheses is that regular post exercise supplementation increase fitness and daily activity level in patients with Fascioscapulohumeral (FSH) muscular dystrophy. All patients are tested before and after 12 weeks of cycle-ergometer exercise. Maximal oxygen consumption and 6MWT is used as primarily effect goals. Secondary effect goals are risk of falls and daily activity level.
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle. To a large extent, patients always lose motor function gradually and die for heart failure or severe infection at the end stage of DMD. At present, the treatment strategy relies on heteropathy accompanied with rehabilitation training. However, the therapeutic effect remains extremely limited. Human umbilical cord mesenchymal stem cells (hUC-MSCs) have been evidenced to improve motor function, increase muscle strength and reduce abnormal levels of related enzymes, such as creatine kinase (CK), lactate dehydrogenase (LDH), alanine aminotransferase (ALT) and aspartate aminotransferase (AST). This study is aimed to explore the safety and efficacy of hUC-MSCs transplantation for DMD.
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.
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.
PDE5A inhibition, which boosts NO-cGMP signaling, will relieve functional muscle ischemia and restore normal blood flow regulation (i.e., functional sympatholysis) during exercise in boys with DMD. The investigators specific aim is to perform an efficient dose-titration study to inform the design of a randomized multicenter trial of PDE5A inhibition for clinical skeletal muscle and cardiac endpoints.