Nusinersen is an antisense oligonucleotide drug that modulates pre–messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA).
“The data published in The New England Journal of Medicine further emphasize the benefit SPINRAZA can provide to individuals with SMA, as the first and only approved SMA treatment in multiple countries. At the end of the ENDEAR study, most infants receiving SPINRAZA showed meaningful benefit, regardless of their age or stage of the disease,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “We are incredibly grateful for the support of the scientists, clinical investigators, and the individuals and families who participated in the studies and continue to contribute to the largest clinical development program to date for the treatment of SMA.” Biogen