Spinal Muscular Atrophy A (Now) Treatable Neurodegenerative Disease

The last 6 years have brought transformative changes to the treatment of SMA, with three approved therapies that are lifesaving in the infantile period and life-altering across the age spectrum. Newborn screening will allow for the greatest benefit of each therapy, as treatment can be started in the pre-symptomatic period. Still, many patients will continue to have residual motor disability, so adjunctive therapies, combined therapies, and improved delivery of current treatments to motor neurons will be needed. The achievements in treating this neurodegenerative disease offer hope for other, more rare forms of SMA, and for neurological diseases across the lifespan.