Nusinersen (Spinraza) is an FDA-approved medication used in the treatment of spinal muscular atrophy, a rare neuromuscular disorder. It is an antisense oligo(ribo)nucleotide (ASO) that is able to induce survival of motor neuron (SMN) expression. In December 2016, it became the first approved drug used in treating this disorder. Nusinersen cost US$750,000 in the first year and US$375,000 annually after that in the United States as of 2019.
纯度:≥98%
CAS:1258984-36-9