Ongoing (Closed To New Participants)


  • Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

    Brief Description: The purpose of the research study is to test an angiotensin converting enzyme (ACE) inhibitor, lisinopril, and an antioxidant, coenzyme Q10 (CoQ10), to ameliorate the decline in cardiac muscle function that occurs in muscular dystrophies. To learn more about the study eligibility and participating sites please visit: https://clinicaltrials.gov/ct2/show/NCT01126697.
  • Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset Facioscapulohumeral Muscular Dystrophy

    Brief Description: The purpose of this study is to establish a standardized functional testing protocol and measure longitudinal changes in muscle strength and function among patients with infantile onset FSHD, to describe the longitudinal changes in clinical phenotypes of infantile FSHD, to evaluate the long-term impact of physical impairment, secondary health conditions, activity limitations and disability caused by FSHD on health-related quality of life and disability, and to evaluate genetic modifiers and biomarkers of clinical phenotypes and disease progression in infantile FSHD. For more information, see the study listing at the following link: https://clinicaltrials.gov/ct2/show/NCT01437345.
  • Becker Muscular Dystrophy- A Natural History Study to Predict Efficacy of Exon Skipping

    Brief Description: The purpose of this study is to understand the disease progression and quality of life in patients with BMD. The information collected will help us understand the clinical progression and quality of life that would result from treating individuals with DMD with exon skipping therapies. To learn more about the study eligibility and participating sites please visit: https://clinicaltrials.gov/ct2/show/NCT01539772.