Methods Of Treating Or Preventing Spinal Muscular Atrophy

A treatment regimen for spinal muscular atrophy (SMA) that rapidly achieves and maintains higher concentrations of antisense compound in patients with SMA. The regimen involves administering a loading dose of the antisense compound at a concentration of 50 mg/mL followed by maintenance doses of 28 mg/mL. This approach enables patients to achieve therapeutic levels of the antisense compound earlier than the approved 12 mg dose of nusinersen, thereby enhancing treatment efficacy. The regimen can be administered intrathecally or intrathecal bolus injections.