The Fault, Dear Neurologist, is Not in Our Genes

The Fault, Dear Neurologist, is Not in Our Genes Neuromuscular medicine has advanced the frontier of neurologic therapeutics.In addition to increasingly specific treatments for immune-mediated neuromuscular disease, the field has led our specialty in disease-modifying therapies for genetic disorders.Vector-based and antisense oligonucleotide therapies are in use for patients with spinal muscular atrophy, leading to entirely new natural histories of this previously fatal disorder.An antisense oligonucleotide has been approved for patients with amyotrophic lateral sclerosis caused by SOD1 variations.In the long view of history, the last decade of therapeutic innovation for neuromuscular disorders has been extraordinary.