View clinical trials related to Muscular Dystrophies.
Filter by:The Synchron Motor Neuroprosthesis (MNP) is intended to be used in subjects with severe motor impairment, unresponsive to medical or rehabilitative therapy and a persistent functioning motor cortex. The purpose of this research is to evaluate safety and feasibility. The MNP is a type of implantable brain computer interface which bypasses dysfunctional motor neurons. The device is designed to restore the transmission of neural signal from the cerebral cortex utilized for neuromuscular control of digital devices, resulting in a successful execution of non-mechanical digital commands.
BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly infusions of trehalose or placebo, followed by a 24-week open-label extension period during which all patients will receive weekly infusions of trehalose. Patients will undergo a safety follow-up assessment 4 weeks after their last treatment.
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males with DMD aged greater than or equal to (>=) 2 to lesser than (<) 5 years. The study will comprise of 2 periods (Period 1: 52-week safety and pharmacokinetics [PK], and Period 2: 52-week extension). Participants will be randomized in a 1:1 ratio to one of 2 treatment arms: 0.9 mg/kg deflazacort, and 0.45 mg/kg of deflazacort. A historic control group (which should match the study population as closely as possible) will be used as a comparator to characterize the safety and tolerability of deflazacort.
A cross-sectional study to explore the relationship between clinically assessed pulmonary function test (PFT) measures and transdiaphragmatic (Pdi) measures in Duchenne muscular dystrophy (DMD) as well as to explore the relationship between sniff nasal inspiratory pressure (SNIP) and transdiaphragmatic (Pdi) measures in Duchenne muscular dystrophy.
The objective of this study is to test a set of clinical outcome measures longitudinally in a cohort of OPMD patients to identify ones that show quantifiable change over time as the disease progresses. The investigators' goal is to delineate the natural history of OPMD.
the Investigator will test the hypothesis that short term PDE5A inhibition (with tadalafil) will reduce post-exercise edema by MRI in males with Becker Muscular Dystrophy.
The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients that will involve direct intramuscular injection to the extensor digitorum brevis muscle (EDB).
The relentless progressive process of muscular dystrophy requires extraordinary medical, physical, and emotional care with severe consequences for caring parents (increased stress and diminished social, psychological and physical well-being). Despite the obvious need of support for parents only few and weak data exist regarding efficiency and efficacy of specific interventions supporting parental resilience and coping strategies. The presenting study aims to fill this gap by evaluating the efficacy of a structured self-management training for parents of children with severe progressive muscular dystrophy compared to parents receiving treatment as usual (TAU). In addition, investigators measure established biomarkers of psychosocial stress, such as pro-inflammatory cytokines, which will be used to monitor physiological changes with assumed significance for parental health.
The purpose of this study was to study the effect of stem cell therapy on Limb Girdle Muscular Dystrophy patients.
The purpose of this study was to analyze the effect of autologous bone marrow mononuclear cells in muscular dystrophy.