Here at Lurie Children’s, our research team works tirelessly to bring our patients and families an abundance of clinical research opportunities, including cutting-edge gene therapy clinical trials. Our multidisciplinary team of clinicians and research staff conduct various research projects to help further the groundbreaking change to treatments for children with neuromuscular disorders. We participate in studies funded by the National Institute of Health (NIH), pharmaceutical companies and our own investigator-initiated studies. We hope our research efforts will improve the lives for children and families living with neuromuscular disorders.
Description: The purpose of this study is to collect natural history, biochemical, pathologic and genetic markers in individuals with neuromuscular weakness. This study offers the potential value for improving clinical care of patients and will aid in designing future clinical trials.
Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)