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Despite significant advances in our understanding of muscle function and disease, development of effective therapies for muscle disorders has remained challenging. Contributing to these challenges are the unique structural and functional properties of muscles that are difficult to replicate in vitro, the diverse genetic background of many muscle disorders, and the lack of in vitro functional assays that can be performed under physiologically relevant conditions. CYTOO pioneered MyoScreen™, an in vitro R&D platform that enables testing muscle therapies in primary patient-derived skeletal muscle cells. Here we describe the development of quantitative cell profiling assays that use myotubes derived from healthy and patient donors to evaluate the activity of therapies targeting muscle disorders in vitro. By analyzing the images of myotubes labeled for selective, disease-dependent markers using machine learning algorithms, we derive profiles that phenotypically distinguish myotubes from healthy and patient donors. AI-powered cell profiling is generally used for the identification and validation of therapeutic targets and biomarkers. By utilizing the profiles determined for healthy and patient donors to define a Health-Score™ (% phenotypically healthy cells out of total), we demonstrate that cell profiling can also be applied to quantitatively assess the ability of therapies to revert disease-associated phenotypes in patient-derived myotubes. This application is particularly useful for the evaluation of exon skipping and gene therapies targeting DMD that lead to the expression of a structurally impaired dystrophin. By modifying the proteins or cellular features used for imaging, our cell profiling approach is applicable to a wide variety of muscle disorders. [ABSTRACT FROM AUTHOR]