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Ublituximab In Autoantibody Positive Immune Mediated Necrotizing Myopathy
Rochester, MN
This is a multi-center, Phase 2 trial of ublituximab as first-line combination therapy in early, active autoantibody positive immune-mediated necrotizing myopathy.
The primary objective is to estimate the effect of ublituximab as first add-on combination therapy at 24 weeks compared to placebo in treating early, active autoantibody positive immune-mediated necrotizing myopathy using the validated 2016 American College of Rheumatology/ European League Against Rheumatism (ACR/EULAR) Myositis Response Criteria, as measured by the Total Improvement Score (TIS)
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Development of Skeletal Muscle Fibers from Patient-Derived Induced Pluripotent Stem Cells (iPSCs)
Rochester, MN
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.
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A Study To Evaluate ASP0367 In Participants With Primary Mitochondrial Myopathy (MOUNTAINSIDE)
Scottsdale/Phoenix, AZ; Jacksonville, FL; Rochester, MN
The purpose of this study is to evaluate the effectiveness, safety and tolerability of ASP0367 in participants with Primary Mitochondrial Myopathy (PMM).
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Nicotinamide Riboside (NR) for Mitochondrial Myopathy Disorder Treatment
Rochester, MN
This study is a phase II, single-center, randomized, double blind, placebo-controlled trail of the efficacy of Nicotinamide Riboside, in the treatment of adult-onset Mitochondrial Myopathy.
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A Study to Evaluate Electrical Impedance Myography as an Outcome Measure in Myositis
Jacksonville, FL
The purpose of this study is to assess Electrical Impedance Myography (EIM) as an outcome measure in patients with Idiiopathic Inflammatory Myopathies (IIM) or Myositis.
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A Study of Muscle Strength in Individuals on Prednisone Treatment that are Having Muscle Weakness
Rochester, MN
The overall main purpose of this investigation is to characterize the electromechanical pathophysiology of the skeletal muscle in individuals with glucocorticoid-induced myopathy.
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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.