Biogen to Run Phase IV SMA Trial: “I Want to Optimize Their Life.”

There are currently three approved therapies for Spinal Muscular Atrophy (SMA). They are Biogen’s Spinraza (nusinersen), Genentech’s Evrysdi and Novartis’ Zolgensma (onasemnogene abeparvovec). The three therapies have been revolutionary in treating SMA, a rare, autosomal recessive neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord. This results in progressive muscle weakness and paralysis. SMA type I, the severest form, was almost always fatal by two years of age, with a 50% mortality rate by seven months and a 90% mortality rate by 12 months, although that has changed with the advent of these therapies.