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Muscular Dystrophy Clinical Trials

A listing of Muscular Dystrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (27) clinical trials

Clinical Trial Readiness for the Dystroglycanopathies

Muscular dystrophies are a diverse group of inherited disorders characterized by progressive muscle weakness and wasting. The disorders are caused by mutations, or changes, in genes. Genes are tiny pieces of inherited material (DNA) that direct the body to make certain kinds of proteins. In this study, researchers will examine ...

Phase N/A

Molecular Analysis of Patients With Neuromuscular Disease

Our research has many goals, one of which is to characterize the genetic changes responsible for the type of muscle disease found in our participants. In our past research, several new genes responsible for various forms of neuromuscular disease were identified and/or are being studied. These include dystrophin, the sarcoglycans, ...

Phase N/A

The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy

The life span in patients with Duchenne muscular dystrophy has been extending due to the development of artificial respiratory devices. According to that, the ratio of cardiac dysfunction as a cause of death has been increasing. This cardiac dysfunction was associated with minor cardiac damage as indicated by elevation of ...

Phase

Evaluation and Diagnosis of People With Pain and Fatigue Syndromes

This screening protocol is designed to facilitate patient recruitment to the National Institute of Nursing Research (NINR) clinical research studies on pain and fatigue syndromes. This protocol will also validate the newly developed Saligan Fatigue Questionnaire. Patients must meet the specific requirements of an IRB-approved research study; this protocol serves ...

Phase N/A

Ramipril Versus Carvedilol in Duchenne and Becker Patients

This protocol represent an open randomized and prospective trial, designed to answer the specific question regarding the role of the cardioprotective therapy in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy patients. In this light, CMR could provide relevant data, reinforcing the scientific background, to start early (particularly in BMD patients ...

Phase

Genetics of Cardiovascular and Neuromuscular Disease

We are studying the genetics of human cardiovascular and neuromuscular disease. There are many different genetic regions that have been associated with the development of cardiomyopathy. An equal number of genetic regions have been associated with muscular dystrophy and there is overlap because some of the identical genes, when mutated, ...

Phase N/A

Molecular and Genetic Studies of Congenital Myopathies

The Congenital Myopathy Research Program consists of a group of scientists and healthcare providers all working to better understand the congenital myopathies. We are taking two approaches to reach our research goals. The first involves identifying and describing new genes and proteins involved in the skeletal muscles that allow our ...

Phase N/A

Study of SRP-4045 and SRP-4053 in DMD Patients

This is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible patients with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 mg/kg SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active ...

Phase

Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy

This study is to determine whether physiologic measures (peak cough flow, measures of respiratory muscle strength including MIP, MEP ,SNIP, and spirometry) can predict spontaneous cough clearance (as measured by a nuclear medicine study) in children with neuromuscular disease. It will also determine whether airway clearance is augmented by high ...

Phase N/A

Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy

This is a multicenter, observational study of patients receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 200 patients across ~50 care centers in Europe and other regions over a period of ~ 2 years. The study population will include patients who ...

Phase N/A