{Reference Type}: Journal Article
{Title}: Clinical perspectives: Treating spinal muscular atrophy.
{Author}: McPheron MA;Felker MV;
{Journal}: Mol Ther
{Volume}: 32
{Issue}: 8
{Year}: 2024 Aug 7
{Factor}: 12.91
{DOI}: 10.1016/j.ymthe.2024.06.020
{Abstract}: Spinal muscular atrophy is a rare and progressive neuromuscular disease that, without treatment, leads to progressive weakness and often death. A plethora of studies have led to the approval of three high-cost and effective treatments since 2016. These treatments, nusinersen, onasemnogene abeparvovec, and risdiplam, have not been directly compared and have varying challenges in administration. In this review, we discuss the evidence supporting the use of these medications, the process of treatment selection, monitoring after treatment, the limited data comparing treatments, as well as future directions for investigation and therapy.