View clinical trials related to Muscular Atrophy.
Filter by:This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age. Cohort 1 is a double-blind placebo-controlled randomized intention to treat protocol for SMA "sitters" 2 - 8 years of age. Cohort 2 is an open label protocol for SMA "standers and walkers" 3 - 17 years of age to explore responsiveness of efficacy outcomes. Outcome measures will include blood chemistries, functional testing, pulmonary function testing, electrophysiological evaluations, PedsQL quality of life assessment, quantitative assessments of survival motor neuron (SMN) mRNA from blood samples, growth and vital sign parameters. Six centers will enroll a total of 90 patients.
Spinal muscular atrophy (SMA) is a disorder that affects the motor neurons. SMA is caused by a mutation in a part of the DNA called the survival motor neuron (SMN1) gene, which normally produces a protein called SMN. Because of their gene mutation, people with SMA make less SMN protein, which results in the loss of motor neurons. SMA symptoms may be improved by increasing the levels of SMN protein. The purpose of this study is to determine whether a drug called a histone deacetylase inhibitor can increase SMN levels. After undergoing a general medical and neurological evaluation, study participants will donate a blood sample. Researchers will use this sample to measure SMN levels. They will also isolate cells from the blood and treat the cells with various drugs that may increase SMN levels.
Patients who are forced to rest or reduce activity as a result of illness, injury, or surgery often experience resulting muscle weakness. This study will evaluate muscle features and muscle strength in patients who are recovering from surgery for broken ankles. The goal of this study is to improve the recovery of muscle function and overall ability after prolonged periods of ankle inactivity due to surgery.
This study's primary objective is to determine the independent and combined effects of progressive resistance muscle strength training and testosterone on the development of sustainable improvements in physical function.
Decreased skeletal muscle mass is a prevalent condition among the elderly, and an important cause of disability and functional decline. The declines in muscle mass associated with aging may be related to alterations in specific kinds of growth factors in the muscle. Elderly hip fracture patients often have significant decreases in muscle mass. The purpose of this study is to investigate whether an exercise program can induce changes in muscle growth factors that are associated with increases in muscle mass and strength in elderly hip fracture patients.
OBJECTIVES: I. Evaluate the effects of androgen suppression with leuprolide and androgen replacement with testosterone enanthate on muscle strength in men with Kennedy's disease or other motor neuron disease.
OBJECTIVES: I. Examine the interrelationships between muscle wasting (phenotype), the degree of myotonic dystrophy (DM) gene expression (genotype) in patients with DM. II. Characterize the insulin resistance in these patients. III. Assess the glucose uptake in the leg and forearm tissues of these patients. IV. Determine the stability of the DM gene lesion in muscles over a 5-10 year period.