Targeted Treatments for Inherited Neuromuscular Diseases of Childhood

Fay AJ, Knox R, Neil EE, Strober J. Targeted Treatments for Inherited Neuromuscular Diseases of Childhood. Semin Neurol 2020;40:335-341   Abstract In the past decade, the number of genes linked to neuromuscular diseases of childhood has expanded dramatically, and this genetic information is forming the basis for gene-specific and even mutation-specific therapies. At the forefront […]