Muscular dystrophy: Research Studies

8 Related Result(s)

  1. Randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy EPIDYS (Epigenetic Rescue of Dystrophin Dysfunction)

    The main purpose of this study is to learn how well the investigational drug givinostat works and how safe givinostat is compared to placebo in males 6 years...

  2. A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy

    The purpose of this research study is to evaluate a new investigational product called “SGT-001” which is designed to target the underlying cause of DMD in...

  3. A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

    We are doing this research study to find out if RO7239361 (previously known as BMS-986089) can help people with Duchenne. We also want to find out if...

  4. Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

    The purpose of this research study is to determine the potential of magnetic resonance imaging to monitor disease progression and to serve as an objective...

  5. Failed Regeneration in the Muscular Dystrophies: Inflammation Fibrosis and Fat

    The purpose of this study is to learn the possible changes that occur in muscles of the lower leg and heart in persons with Muscular Dystrophy. The goal is...

  6. Brain imaging and Social cognition in myotonic dystrophy (DM) type 1

    This study is designed to assess social cognition deficits in adult onset patients with myotonic dystrophy type 1 and understand possible neuroimaging...

  7. Studies of skeletal muscle and gastrointestinal dysfunction in myotonic dystrophy and controls

    This study is designed to obtain data regarding 2 aspects of the phenotype in myotonic dystrophy (dystrophia myotonica or DM). These are multi-system...

  8. Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

    This study aims to help drug developers successfully design clinical trials and understand how to assess the efficacy of potential therapies. Researchers are...