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.
Found 135 clinical trials
phenotypeS in Non Ambulant Duchenne Muscular Dystrophy
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
Spanish Natural History Study for LAMA2 Muscular Dystrophy
The objective of this natural history study is to comprehensively characterize the disease progression and clinical features of LAMA2-related dystrophies (LAMA2-RD) in the pediatric population. The study aims to establish a well-defined cohort of patients in Spain, enabling long-term follow-up and facilitating recruitment for future clinical trials.
Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
The goal of this observational study is to learn the patient journey, disease burden, living situation, quality of life and etc. in patients with Facioscapulohumeral Muscular Dystrophy in all sex/gender and age groups. The main questions it aims to answer are: What's the patient journey of patients with Facioscapulohumeral Muscular …
Phenotype - Genotype Correlation in a Sample of Egyptian Patients With Congenital Myopathies and Congenital Muscular Dystrophies
The aim of this study is to correlate the phenotype and genotype among a sample of Egyptian patients with Congenital myopathies and Congenital muscular dystrophies.
Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
The aim of this study is to verify whether full video home polysomnography (sleep study) with nocturnal transcutaneous CO2 monitoring is feasible in the follow-up of non-invasive ventilation (NIV) in patients with Duchenne muscular dystrophy. The researchers would like to investigate whether they could perform sufficient measurements in the home …
Oral Health, Saliva Viscosity and Composition in Oculo-Pharyngeal Muscular Dystrophy (OPMD)
The goal of this observational study is to explore whether oral health and saliva viscosity and composition in Oculopharyngeal Muscular Dystrophy (OPMD) is different from control subjects. The main questions it aims to answer are: Do swallowing disturbances in OPMD adversely affect oral health? Is saliva thickness (viscosity) is increased …
Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry
The goal of this open prospective multi-disciplinary observational study of patients with OPMD at various stages of clinical manifestations is to explore the natural history of the disease in the Israeli population.
Estimation of Non-Reimbursable Costs for Patients With Duchenne Muscular Dystrophy in France
Duchenne muscular dystrophy (DMD) is a chronic neuromuscular disorder affecting approximately 150 to 200 newborns annually, predominantly males, and is characterized by progressive muscle atrophy and weakness due to a complete absence of dystrophin. DMD presents a severe phenotype, with life expectancy typically extending into the third decade. Data on …
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems. Participants will undergo: Neuromuscular assessments Blood collections Swallowing and breathing assessments Questionnaires
A 5-year Natural History Study in LAMA2-related Muscular Dystrophy and SELENON-related Myopathy.
SELENON-related myopathy (SELENON-RM) and LAMA2-related muscular dystrophy (LAMA2-MD) are congenital neuromuscular disorders presenting with slowly, progressive axial muscle weakness, spinal rigidity, scoliosis and respiratory insufficiency. Currently, no curative treatment options exist, yet promising preclinical trials are ongoing. Clinical trials are expected to start within 5 years. Natural history data and …
