New Therapeutics Options for Pediatric Neuromuscular Disorders.
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ABSTRACT:
SUBMITTER: Flotats-Bastardas M
PROVIDER: S-EPMC7719776 | biostudies-literature | 2020
REPOSITORIES: biostudies-literature
Publications
New Therapeutics Options for Pediatric Neuromuscular Disorders.
Flotats-Bastardas Marina M Hahn Andreas A
Frontiers in pediatrics 20201123
Neuromuscular disorders (NMDs) of Childhood onset are a genetically heterogeneous group of diseases affecting the anterior horn cell, the peripheral nerve, the neuromuscular junction, or the muscle. For many decades, treatment of NMDs has been exclusively symptomatic. But this has changed fundamentally in recent years due to the development of new drugs attempting either to ameliorate secondary pathophysiologic consequences or to modify the underlying genetic defect itself. While the effects on ...[more]
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