An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen...
Spinal Muscular AtrophyThis study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with spinal muscular atrophy (SMA). Four dose levels will be evaluated sequentially. Each dose level will be studied in a cohort of approximately 8 participants, where all participants will receive active drug.
Effect of GTx-024 on Muscle Wasting in Patients With Non-Small Cell Lung Cancer (NSCLC) on First...
Muscle WastingNon-Small Cell Lung CancerThe purpose of this study is to determine if the investigational drug GTx-024 can help patients with non small cell lung cancer increase physical function and maintain or gain muscle.
Effects of Inspiratory Muscle Training on Dyspnea Perception During Exercise in Patients With COPD...
COPDSevere Systemic Illness-induced Respiratory Muscle WastingPatients with chronic obstructive pulmonary disease are often limited in their exercise capacity by intolerable shortness of breath (dyspnea). Patients are breathing at high lung volumes during exercise which forces inspiratory muscles to work at a high percentage of their maximal capacity. This increased inspiratory effort has been shown to be independently related to symptoms of dyspnea during exercise in previous research. Eight weeks of high intensity variable flow resistive inspiratory muscle training is hypothesized to reduce inspiratory effort and to decrease neural drive to inspiratory muscles. These factors are hypothesized to jointly contribute to delaying the occurrence of intolerable symptoms of dyspnea and to improve exercise tolerance in these patients.
Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
Spinal Muscular Atrophy Type 3The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).
Effects of Inspiratory Muscle Training in Elders
Muscular AtrophySensitivity Training Groups1 moreAging causes anatomical and physiological changes in the respiratory system and respiratory muscle strength with decline in its maximum function. This study aimed to evaluate the effects of inspiratory muscle training on respiratory muscle strength, thickness of the diaphragm and diaphragmatic mobility in older women. The investigators' hypothesis is that inspiratory muscle training improves respiratory muscle strength, the thickness of the diaphragm and diaphragmatic mobility in older women.
Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients...
Muscular AtrophySpinalA two-period, two-treatment, crossover study to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ambulatory patients diagnosed with spinal muscular atrophy (SMA) Type 3.
Anabolic Effects of Intraoperative Feeding in Reconstruction Surgery
FastingFeeding7 morePerioperative fasting remains a common clinical practice in surgical patients to prevent the development of postoperative anesthesia- and surgical-related complications. Clinical observational studies indicated that the combination catabolic effects resulted from prolonged perioperative fasting and profound surgical stress are likely to induce extensive protein catabolism, muscle breakdown and impaired glycemic control during postoperative phase, leading to the development of severe complications. Furthermore, prolonged gastrointestinal fasting is associated with microbial translocation that deteriorates the early recovery after surgery. This clinical trial anticipates in determining the beneficial effect of intraoperative feeding to improve intraoperative hemodynamics and enhance postoperative recovery due to attenuation of systemic catabolism and improvement of insulin sensitivity to glycemic control.
A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated...
Spinal Muscular AtrophiesA long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Effects of Ketones on Muscle Wasting During Caloric Restriction in Lean Females
Muscle WastingProtein Intake2 moreBecause of these anabolic properties of ketone bodies and the fact that ketone bodies prevent muscle protein breakdown for gluconeogenesis during energetic stress, ketone bodies can be a promising strategy to prevent or treat skeletal muscle wasting. Therefore, our aim is to investigate the effect of 3HHB intake on muscle wasting and its adverse consequences during a period of caloric restriction in lean females. In addition, we compare the effects of 3HHB intake with a high protein diet, which is currently considered as the best strategy to minimize lean loss during hypo-energetic periods. To end, we aim to investigate the synergistic effects of the intake of 3HHB in combination with a high protein diet.
Effect of Branched-chain Amino Acid Supplementation and Exercise on Muscle Quantity and Quality...
Muscle AtrophyIntroduction: Ultrasound can be used to monitor muscle mass during interventional approaches in patients with liver cirrhosis. The aim: To investigate the effect of branched-chain amino acid (BCAA) supplementation and/or muscle exercise on ultrasound-measured quadriceps muscle thickness and echo intensity, as well as on muscle strength, performance, and nutritional assessment in patients with cirrhosis. Patients & Methods: This is a randomized controlled study that included 220 liver cirrhosis patients with Child-Pugh B & C classes. They were randomized into a control group comprising 55 patients who received only the standard care, and interventional groups comprising 165 patients equally distributed into three groups, they received in addition to standard care, BCAA, programmed exercise, or BCAA and programmed exercise respectively. At baseline and after 28 days, all participants were subjected to ultrasound-measured quadriceps muscle thickness and echo intensity, in addition to handgrip strength, short physical performance battery (SPPB), anthropometric measures, hematological and biochemical assessment, MELD score measurement, nutritional assessment using 7- subjective global assessment score (SGA).