Muscle biopsy: Research Studies

2 Related Result(s)

  1. 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...

  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...