Effects of Neuromuscular Electrical Stimulation on Muscle Mass and Strength in Critically Ill Patients...
Neuromuscular Electrical StimulationIntensive Care Unit Acquired Weakness2 moreThe purposes of this study are 1) to determine whether neuromuscular electrical stimulation (NMES) is effective in preventing loss of muscle mass and strength and 2) to observe the time variation of MLT and strength from preoperative day to hospital discharge.
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants...
Spinal Muscular AtrophyThe primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA). The secondary objectives are to examine the safety and tolerability of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA and to examine the cerebral spinal fluid (CSF) and plasma Pharmacokinetics (PK) of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA.
Preventive Effect of Proprioceptive Stimulation on Muscle Atrophy
Knee ArthritisConfinement to bed, which occurs in many pathological situations, induces a muscle atrophy and a loss of muscle strength. Muscle atrophy is associated to impaired performance in motor tasks, such as posture and locomotion, and is therefore a major cause of loss of autonomy. It requires a stay in follow-up and rehabilitation service, and thus lengthens the duration of hospitalisation. Data underline the importance of afferent input integrity in the maintenance of muscle characteristics and postural control, and suggest that countermeasure programs based on the stimulation of proprioceptive inputs could be efficient to prevent muscle atrophy and falls. In particular, fundamental studies performed in rodents by the investigators laboratory have demonstrated that the adverse structural and functional adaptations which occur during muscle deconditioning can be counteracted through adequate physiological stimuli such as activation of proprioceptors. Based on this scientific expertise, the investigators aim is thus to prevent muscle atrophy and its functional consequences on posture and locomotion, following a surgical intervention in humans . The investigators will develop a device allowing stimulation of foot and ankle proprioceptors. In order to facilitate the evaluation of its efficiency, the device will be tested on a selected population confined to bed during a post-operative period (knee replacement). It efficiency will be evaluated by means of three parameters: muscle force of ankle plantar flexor, muscle volume of lower limb muscles, functional outcome (gait and balance analysis). The technique developed in the present project could bring benefits to patients confined to bed, or in elderly. Preventing or retarding development of muscle atrophy will beneficiate to health and quality of life of these patients. In addition, this device might allow to consider therapeutic strategies for prevention of atrophy in neuromuscular pathologies.
Testosterone Administration and ACL Reconstruction in Men
Muscle AtrophyACL Reconstruction2 moreThis study is being done to test whether taking testosterone can prevent loss of muscle mass and strength due to anterior cruciate ligament (ACL) reconstructive surgery. Testosterone is the principal male sex hormone and an anabolic (muscle promoting) steroid. It is essential for the development of male reproductive tissues and promotes increased muscle, bone mass, and the growth of body hair. The investigators hope to learn whether testosterone given before and after ACL reconstructive surgery will increase muscle mass and strength and potentially improve recovery time following surgery.
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
Spinal Muscular Atrophy 1The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
Progressive Strength Training in Spinal Muscular Atrophy
Muscular AtrophySpinalThe proposed feasibility study is necessary to test if children and young adults will participate in and adhere to a 12-week, home-based, supervised progressive strength training exercise program and to obtain preliminary data that will subsequently allow us to determine the safety and impact of strength training in spinal muscular atrophy. Our pilot study will address 3 aims: (1) Ascertain the feasibility of, and potential barriers to, participation in and adherence to a 12-week home-based, supervised, progressive strength training exercise program in children and young adults aged 5-21 years with SMA types II and III; (2) Determine the safety and tolerability of progressive strength training in a pilot study sample of children and young adults with SMA types II and III; and (3) Determine candidate outcome measures.
Effects of Power Mobility on Young Children With Severe Motor Impairments
Cerebral PalsyArthrogryposis2 moreThe purpose this study is to determine the effects of power mobility on the development and function of young children of young children whose severe physical disabilities limit their exploratory behaviors and may unnecessarily restrict their cognitive, communication, and social-emotional development.
Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy...
SMAThis is a multicentric, randomized, double-blind study versus placebo, with two parallel groups treated to evaluate the efficacy and the tolerance of Riluzole in children and young adults (6 to 20 years of age) with SMA. (Type II and Type III).
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
Spinal Muscular AtrophyNeuromuscular DiseaseThis study is being conducted to test whether exercise can be effectively used as an intervention to treat Spinal Muscular Atrophy (SMA). In order to answer this question, the investigators will enroll 14 subjects with SMA between ages 8 and 50 and ask them to complete an 18 month training schedule. At some points subjects will be asked to closely follow a specific training regimen and at other points they may be asked to exercise in the same manner they do normally. The exercises they will be asked to perform include biking on a stationary cycle and lifting hand weights. Subjects will be asked to come in to the clinic seven times over the course of the study to perform tests. These tests include motor function measures, a physical exam, questionnaires, a exercise capacity test which involves riding a stationary bicycle, and test where the subject is asked to walk as far as they can in six minutes. The main goal of the study is to see if the subjects who participate in the exercise protocol have larger increases in the distance they can walk in six minutes than those who do not.
Phase III Study of the Effect of GTx-024 on Muscle Wasting in Patients With Non-Small Cell Lung...
Muscle WastingNon Small Cell Lung CancerThe purpose of this study is to determine if the investigational drug GTx-024 can help subjects with non-small cell lung cancer increase physical function and maintain or gain muscle, also called "lean body mass".