Muscular dystrophy

Displaying 4 studies

  • A Study to Evaluate Single DNA Molecule Optical Imaging to Diagnose Facioscapulohumeral Muscular Dystrophy (FSHD) Rochester, MN

    The purpose of this study is to evaluate a novel direct single DNA molecule optical imaging system (Bionano) for the FSHD diagnosis.

  • Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy Rochester, MN

    The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.

  • A Study to Create a Neuromuscular Observational Research (MOVR) Data Hub Rochester, MN

    MOVR Data Hub is the first and only data hub that aggregates clinical, genetic and patient reported data for multiple neuromuscular diseases. The combination of data collected through MOVR Data Hub will provide a comprehensive understanding of health and patient experiences in a single data repository. MOVR Data Hub has the power to transform health outcomes, accelerate drug development and drive clinical research across multiple neuromuscular diseases, helping to ensure that the next generation of individuals living with neuromuscular disease are afforded every opportunity to thrive.

  • Inherited Muscle Diseases Repository Rochester, MN

    To establish a repository of DNA samples collected prospectively from patients with inherited myopathies of unknown molecular and/or biochemical defect. To access residual muscle specimens from diagnostic muscle biopsies obtained as part of the routine medical care. The biospecimens will be used to better understanding the underlying molecular defects and mechanisms of muscle diseases.


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