Patent ID: 8980853
Filing Date: 2015-03-17
Classification: A61K,A61P,C12N

Abstract:
1. A method comprising administering by a bolus injection into the intrathecal space of a subject with infantile-onset type I spinal muscular atrophy (SMA) an antisense compound comprising an antisense oligonucleotide consisting of 18 linked nucleosides, wherein the oligonucleotide has a nucleobase sequence consisting of the nucleobase sequence SEQ ID NO: 1, wherein each internucleoside linkage of the oligonucleotide is a phosphorothioate linkage, wherein each nucleoside of the oligonucleotide is a 2′-MOE nucleoside, and wherein the administering of the antisense compound ameliorates at least one symptom of SMA in the subject.