This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Muscular dystrophy (MD) comprises a group of distinct inherited degenerative muscular disorders characterized by progressive muscle weakness and wasting of variable muscle groups with variable severity. The genes and protein products that cause Becker muscular dystrophy (BMD) and most forms of limb-girdle muscular dystrophy (LGMD) disorders have been identified, and this information helps establish an accurate diagnosis. In facioscapulohumeral muscular dystrophy (FSHD), a chromosome 4q deletion is highly specific for diagnosis. Available treatment options are inadequate as there is currently no approved drug, cure, or long-term effective treatment for any type of muscular dystrophy The primary objective of this study is to evaluate the safety of MYO-029 in adult subjects with MD. Secondary Objectives: To evaluate the biological activity of MYO-029 in adult subjects with MD by using the following measurements: muscle mass, volume, strength; histopathology findings; patient-reported outcomes; and functional measurements. To explore associations between gene expression patterns, test article administration, and clinical parameters. To evaluate the pharmacokinetics of MYO-029 in adult subjects with MD
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