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  • Development of Skeletal Muscle Fibers from Patient-Derived Induced Pluripotent Stem Cells (Ipscs) to Study Mechanisms of Hereditary Myopathies Rochester, Minn.

    The goal of this study is to create a repository of skin fibroblasts from patients with inherited myopathies or suspected inherited myopathies. The study will optimize the development of skeletal muscle cells from patient induced pluripotent stem cells (iPSCs) and to characterize their morphological, biochemical, electrophysiological and molecular properties. The study will also use skin fibroblasts as source of DNA to investigate for research purpose the genetic defect causing the myopathy, and test in future potentially therapeutic drugs in the iPSC-derived muscle cells.

  • Inherited Muscle Diseases Repository Rochester, Minn.

    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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  • Neuromuscular Observational Research (MOVR) Data Hub (MOVR) Rochester, Minn. 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.

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