Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy.

ABSTRACT: BACKGROUNDSpinal muscular atrophy (SMA) is a rare genetic neuromuscular disease caused by deletions or mutations of the survival motor neuron 1 (SMN1) gene. Despite the availability of genetically based treatments for SMA, functional impairments and weakness persist in treated symptomatic individuals. This study addresses whether additional treatment after gene transfer therapy could provide further clinical benefits.METHODSInterim day 302 findings are described from the phase IV open-label RESPOND trial evaluating nusinersen in participants aged ≤36 months who had suboptimal clinical status following onasemnogene abeparvovec (OA) treatment, as determined by the investigator.RESULTSThirty-seven participants included in the interim analysis were symptomatic at the time of OA administration.

SUBMITTER: Proud CM 

PROVIDER: S-EPMC12618081 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature