Reducing Disease Burden Through Early Diagnosis and Treatment of Spinal Muscular Atrophy (SMA)

Dennis P. Scanlon, PhD; Emma Ciafaloni, MD; Maria Lopes, MD, MS; Kevin U. Stephens, Sr, JD, MD; and Mary Schroth, MD, FAAP, FCCP provide an overview of the disease burden in spinal muscular atrophy and give insight into clinical and payor perspectives on screening and diagnosis, current treatment options, and new disease-modifying therapies in SMA.