Active clinical trials for "Syndrome"

The purpose of the study is to evaluate the safety and efficacy of two dose levels of Naglazyme in infants under the age of one year who have MPS VI by monitoring physical appearance, x-ray of the skeletal system and growth.

RATIONALE: Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. PURPOSE: Phase II trial to study the effectiveness of monoclonal antibody therapy in treating patients who have primary myelodysplastic syndrome.

Background: Recent studies indicate that exercise-related games can improve executive function, attention processing, and visuospatial skills. Objective: This study investigates whether exercise with exergaming can improve the executive function in patients with metabolic syndrome (MetS). Methods: Twenty-two MetS patients were recruited and randomly assigned to the exergaming group (EXG) and treadmill exercise group (TEG). The reaction time (RT) and electrophysiological signal from the frontal (Fz), central (Cz), and parietal (Pz) cortex were collected during a Stroop task after 12 weeks' exercise.

This is a placebo-controlled clinical trial to assess the utility of Guanfacine Extended Release (GXR) in the management of patients with Prader Willi Syndrome (PWS) who have significant aggression or self-injury. The purpose of this trial is to establish the safety of GXR with a specific focus on metabolic effects.

This study assesses the clinical effectiveness of mammalian target of rapamycin (mTOR) inhibition with rapamycin in minimizing or decreasing the severity of acute lung injury/acute respiratory distress syndrome (ALI/ARDS) in participants infected with mild to moderate COVID-19 virus.

It has been suggested that the actual obesity epidemy is related to chronic overconsumption of added or free sugars. The increasing popularity of artificial sweeteners attest the population willingness to reduce added sugars intake and to use alternatives to alleviate health impact of free sugar overconsumption. However, recent findings suggest that artificial sweeteners may rather contribute to obesity epidemy and its associated adverse health effects, potentially via a negative impact on gut microbiota. It has been shown in various studies that, for the same amount of sucrose, unrefined sugars (such as maple syrup) are associated with favorable metabolic effects. The polyphenols contained in maple syrup, especially lignans, could contribute to these positive effects. Indeed, the strong impact of those biomolecules on the modulation of gut microbiota and on gastro-intestinal and metabolic health has been demonstrated in several studies. It is therefore highly relevant to test the hypothesis that the substitution of refined sugar by an equivalent amount of maple syrup (5% of daily energy intake) result in a lesser metabolic deterioration, by the modulation of maple syrup on gut microbiota, than the one observed with refined sugar.

The primary objective of this study is to monitor the long-term safety of DCCR in PWS patients.

Down syndrome is the most common genetic disorder in the society that causes mental retardation. Today, screening tests (combined test, triple screening, ultrasonography and age) are performed for the diagnosis of down syndrome for all pregnant women. As a result of screening tests, amniocentesis is performed as a diagnostic test for the group at risk. Chromosome analysis from amniotic fluid requires a 3-week period for chromosome cultures to yield results. Several levels of biochemical markers, such as organic acids and pyridoxine metabolites, have been found to be elevated in the amniotic fluid. The investigators also plan to investigate ischemia-modified albumin, hepatocyte growth factor level in amniotic fluid.

The main objective of the study is to demonstrate that the inclusion of eggs to a plant-based diet will not increase the risk for heart disease but will increase the concentration of lutein and zeaxanthin, two important antioxidants and will also increase choline, an important component of membrane phospholipids.

This is a single center, Phase 1/2 study in which patients with Hurler syndrome who have previously undergone allogeneic hematopoietic stem cell transplantation are treated with autologous plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system.