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We list hundreds of Clinical Trials about "Sarepta Therapeutics playing nice with over rejection Duchenne" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.
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The purpose of this study is to determine if ACE-031 is safe and well-tolerated in children with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for designing future studies.
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
The purpose of this research study is to understand the walking patterns, strength and function changes of boys with Duchenne muscular dystrophy on/off corticosteroids to determine the best timing and treatment options to maintain walking for as long as possible.
The safety objective of the HOPE-Duchenne trial will be to investigate the safety and tolerability of CAP-1002 administered by multi-vessel intracoronary infusion in subjects with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD). The efficacy objective of the HOPE-Duchenne trial will be to investigate the efficacy of CAP-1002 administered by multi-vessel intracoronary infusion in subjects with cardiomyopathy secondary to DMD by stabilizing or improving cardiac str...
1. Comparing different lines of treatment of Duchenne Myopathy (DM) and assessment of new lines of treatment (mesenchymal stem cell, phosphodiesterase inhibitors) in reducing the impact of disability in the patients with Duchenne Myopathy and slowing the progression of cardiomyopathy 2. Upsetting and implementation of the best treatment plan for those children with Duchenne myopathy which is suitable for the available resources in Assiut University Childr...
The study seeks to discover whether peer rejection increases the value of food relative to peer interaction in overweight individuals. After playing a computer game that randomly simulates peer rejection or peer acceptance, participants will play another computer game that will assess the value of food and social interactions. Overweight individuals may be more likely to resort to food in moments of distress and less likely to choose to interact with a peer to reestablish thei...
Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 6 Years over a period of 6 to 24 months using disease appropriate evaluations.
This study evaluates the function of the heart in young patients with muscular dystrophy type Duchenne or Becker. Participants have their hearts examined at regular intervals by ultrasound (echocardiography) and cardiac magnetic resonance imaging.
The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
Data on preventive therapy in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) affected individuals without cardiac involvement are very limited and currently lacking regard both ACE-inhibitors and Beta-Blockers in Becker Muscular Dystrophy and for the latter even in Duchenne Muscular Dystrophy patients. Thus, the study aim is to compare the efficacy of carvedilol vs ramipril on myocardial tissue properties and heart function, performing CMR and myocardial ...
OBJECTIVES: I. Characterize the effect of prednisone on muscle protein metabolism in patients with Duchenne muscular dystrophy. II. Determine whether prednisone changes levels of insulin-like growth factor 1, growth hormone, and insulin. III. Characterize the effect of prednisone on muscle morphometry and muscle localization of utrophin. IV. Compare the prednisone response in patients with Duchenne muscular dystrophy to that seen in normal individuals and in ...
This study examine whether an evidence-based individual user-preferred exercise program will increase the physical activity level in boys with Duchenne muscular Dystrophy (DMD).
The use of topical Cyclosporine A early after an episode of endothelial graft rejection after penetrating keratoplasty and continuing its administration for 6 months can reduce the course of that episode and recurrence of the rejection.
On July 14, 2016, in Nice, children and their families were attacked by the organization "EI". In Nice, 86 deaths, including 10 children, the youngest at 4, were recorded. A number of children, still difficult to assess exactly but over 100, was bereaved. After a traumatic event, multiple clinical consequences may appear in children. Among these consequences, the most common is Post-Traumatic Stress Disorder (PTSD). The aim of the study is to characterize the psycho-social fac...
The main purposes of this study are to see if it is safe to use a new medication called vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see if vamorolone works for the treatment for DMD, and to see how any potential side effects compare to those seen in boys using steroids.
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.
The primary objective of this investigation is to assess the effectiveness of transcutaneous electrical nerve stimulation applied using VECTTOR to reduce the symptoms of Duchenne Muscular Dystrophy and reduce the impact of DMD upon the participants' quality of life. The primary outcome measures will include: 1. increased muscle strength and 2. increased range of joint motions.
The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
The main objective of this study is to evaluate the safety of a high and low dose of NS-065/NCNP-01 injection delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Additional objectives include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.
The purpose of this study was to test the feasibility and preliminary efficacy of playing Pokémon Go to increase daily steps among young adults. The study also aimed to assess people's experience playing the game, identify specific features of the game that users like/dislike, and explore game features that may promote overall well-being.
The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages 4-
Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy. The aim of this study is to determine the clinical spectrum and natural progression of dystrophinopathy in a prospective multicenter natural history study, to assess the clinical, genetic of patients with dystrophinopathy to optimize clinical management.
Development of a new MS-based biomarker for the early and sensitive diagnosis of Duchenne disease from plasma