SMA Moment: Will Gene Therapy Shift Treatment, Costs of Muscle Disease?

This is a big moment for people diagnosed with spinal muscular atrophy, or SMA, a rare and potentially lethal genetic disorder that destroys muscles. For decades, there was no way to change the trajectory of their disease. They now have one marketed medicine, and this month, chances are they’ll have another: a gene therapy that promises a long-lasting treatment, if not an outright cure, through a one-time dose.