Clinical Trials

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.

The purpose of this study is to evaluate the effectiveness, safety and tolerability of ASP0367 in participants with Primary Mitochondrial Myopathy (PMM).

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.

Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM study")

The overall main purpose of this investigation is to characterize the electromechanical pathophysiology of the skeletal muscle in individuals with glucocorticoid-induced myopathy.

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.

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.