SMA Trials
Location Trial Status:
= Fully recruited
= Recruiting
= Not yet recruiting
= Enrolling by invitation
= Completed / Terminated
= Fully recruited
= Recruiting
= Not yet recruiting
= Enrolling by invitation
= Completed / Terminated
An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy.
This is a Phase 2 clinical trial to test the safety and efficacy of Nusinersen in infants with genetically diagnosed and presymptomatic SMA. This study also aims to examine the effects of Nusinersen in infants.
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