AAN 2017: Two clinical studies report promising data for new spinal muscular atrophy treatment

Two studies presented at the AAN Annual Meeting 2017 in Boston (MA, USA) have demonstrated positive results for nusinersen (Spinraza® Biogen (MA, USA) in the treatment of spinal muscular atrophy (SMA). Nusinersen is an antisense oligonucleotide designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q, which lead to SMN protein deficiency. Through alteration of SMN2 pre-mRNA, nusinersen acts to increase production of SMN full-length protein. In the Phase III CHERISH study, 126 children who had experienced SMA symptom onset aged 6 months or older were randomized to nusinersen treatment or placebo control. In...