View clinical trials related to Muscular Diseases.
Filter by:The purpose of this study is to determine the effects of Nicotinamide Riboside (NR) supplement in adult-onset symptoms of mitochondrial myopathy.
This study's purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or certain other subtypes of polymyositis (PM; including antisynthetase syndrome [ASyS]) will be included in the study. Treatment response will be measured by Total improvement score (TIS). Additional information can be found on https://myositis-study.com/.
AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
Quality of life (QoL) assessment is a milestone of clinical practice in gynecology. In particular, the use of validated QoL questionnaires is of the utmost importance for pelvic floor disorders, due to their functional nature and high prevalence. Pelvic floor dysfunctions such as lower urinary tract, bowel and sexual dysfunctions often coexisting with pelvic organ prolapse affect a substantial proportion of women and can often cause bothersome symptoms and have a negative effect on psychological and social well-being. Assessment of QoL in women with pelvic floor dysfunctions is essential for making a diagnosis and designating an adequate treatment. Therefore, it is necessary to evaluate women's subjective perception. A valid way to measure the patients' perspectives is through self-administered questionnaires that can address the presence and severity of symptoms, and their impact in daily activities and QoL. Symptom severity and QoL can be assessed by two specific questionnaires, the Pelvic Floor Distress Inventory (PFDI-20) and the Prolapse Quality of Life (P- QOL).
Endocrine diseases including Cushing's syndrome and phaeochromocytoma/paraganglioma (PPGL) but not Conn's syndrome are associated with muscle wasting and weakness. The study's aim is to identify epigenetic determinants of muscle homeostasis in these conditions following medical treatment and adrenalectomy. This is an observational pilot study that will recruit 66 patients from 3 diagnostic groups: Cushing's syndrome (16), PPGL (20) and Conn's syndrome (30). Indices of muscle bulk and strength will be assessed at diagnosis and at outpatient follow-up 6-9 weeks after adrenalectomy. At these times blood and urine will be collected and a muscle biopsy taken from the operation site at the time of surgery. Pathway analysis in these samples will identify potentially novel signalling pathways contributing to muscle wasting via prolonged exposure to high levels of corticosteroid and catecholamines. This will highlight commonalities and differences in pathogenesis of muscle wasting from a variety of different causes. Finally, it will inform identification of novel therapies for muscle atrophy.
This study aims to explore the clinical characteristics and mechanism of inhibitors of janus kinase in the treatment of idiopathic inflammatory myopathies
Objectives: To observe the effect of newly-created individualized upper airway muscle functional training on the condition and intraday symptoms of OSA patients; to study the effect of this training method on the excitability of the genioglossus muscle cortex; to analyze the factors affecting the efficacy of upper airway training in the treatment of OSA and screening suitable population for upper airway training: Design: A randomized double-blind controlled trial. SAS 9.3 statistical software (SAS Institute, Cary, North Carolina, USA) was used to generate a random number table, and the selected patients were randomly divided into experimental group 1, experimental group 2, and control group according to the ratio of 1:1:1 with 100 cases each. Unit: Shenyang, China Participants: Consecutive specific OSA patients, who are potential candidates for the treatment of upper airway training (n=300), will be recruited from a sleep center or respirologists, psychiatrists, otolaryngologists and dentists practicing with broad inclusion criteria (age: 20-75 years, AHI:15-50/h; BMI<40 kg/m2). Interventions: The three groups of subjects completed 7-day functional training and control training of upper airway muscles in different modes, respectively completed polysomnography, neck circumference, Berlin questionnaire and Epworth sleepiness scale before and after training, The genioglossus myoelectric activity was measured after transcranial magnetic stimulation and the excitability of the genioglossus cortex motor center was used to determine the efficacy of different training. After regression analysis, the factors affecting the efficacy of upper airway muscle group training were analyzed to screen the OSA patient population suitable for upper airway muscle group training.
Positive effect of physical activity on health arouses a strong interest at international level and is developped within the scope of national programs. Recommandations exist but must be designed for patients with functional limitations of activities. Patients with mitochondrial diseases have exercice intolerance with an increase of muscular weakness and fatigue after low exercice volume. Theses patients have functional limitations of activities. In order to establish an appropriate training programme, it will be important to define and consider the physical condition. The Society of Mitochondrial Medecine published recommandations for management of theses patients,However, theses recommandations do not allow them to propose a training program of what can be done. For these vulnerable patients, therapists are responsible fo advising a training programm without guidelines to establish its terms and conditions. In addition, some exercices do not appear to have been the subject of complete assessmeents. Regarding training programs (aerobic training, muscle reinforcement, miwed training), scientific literature shows a significant genetic and clinical variabilities, as well as a lack of data on clinical severity of included patients. In addition, the lack of informations regarding training effects of heteroplamy level limits our comprehension of mechanisms involved in adaptation of mitochondrial pool during training. Therefore, further reserchs on this subject are essential. It is necessary to offer these patients a follow-up and personalized training program, which are in adequation with daily life. Some publications call on specifics concepts which are not compatible with day-to-day life. The investigators think it will be useful to investigate training effects in order to have practival conclusions, easily reproducible at home by patients with simple and inexpensive equipment. In this context, video consultation could allow the close follow-up of these patients. The investigators hypothesize that a mixed training (endurance and muscle reinforcement), personalized, at home and followed by video consultation have positive effects on some physical criteria (such as musclar strength, tolerance to effort, functional abilities) without increasing heteroplasmy and creatine phosphokinase levels.
To evaluate the effects of 3-months annatto-derived geranylgeraniol (GG) supplementation on statin-associated skeletal muscle health.
The purpose of this study is to determine whether patients with acute kidney injury requiring renal replacement therapy have a higher incidence of muscle wasting than controls and whether the course of recovery is longer compared to controls.