Genetic approaches to the treatment of inherited neuromuscular diseases

Abstract excerpt

Inherited neuromuscular diseases are a heterogeneous group of developmental and degenerative disorders that affect motor unit function. Major challenges toward developing therapies for these diseases include heterogeneity with respect to clinical severity, age of onset and the primary cell type that is affected (e.g. motor neurons, skeletal muscle and Schwann cells). Here, we review recent progress toward the...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Muscle Physiology and Disorders
• Share to Neurogenetic and Muscular Disorders Research
• Share to RNA Research and Splicing