New pharmacotherapies for genetic neuromuscular disorders: opportunities and challenges
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DOI: 10.1080/17512433.2019.1634543
Abstract: ABSTRACT Introduction: Genetic neuromuscular diseases (NMDs) constitute a heterogeneous group of rare conditions, including some of the most disabling conditions in childhood. Recently, advanced technologies have greatly expanded preclinical and clinical research, and specific therapies… read more here.
Keywords: neuromuscular disorders; pharmacotherapies genetic; opportunities challenges; disorders opportunities ... See more keywords