Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies.

ABSTRACT:

Objective

To report results of intrathecal nusinersen in children with later-onset spinal muscular atrophy (SMA).

Methods

Analyses included children from a phase 1b/2a study (ISIS-396443-CS2; NCT01703988) who first received nusinersen during that study and were eligible to continue treatment in the extension study (ISIS-396443-CS12; NCT02052791). The phase 1b/2a study was a 253-day, ascending dose (3, 6, 9, 12 mg), multiple-dose, open-label, multicenter study that enrolled children with SMA aged 2-15 years. The extension study was a 715-day, single-dose level (12 mg) study. Time between studies varied by participant (196-413 days). Assessments included the Hammersmith Functional Motor Scale-Expanded (HFMSE), Upper Limb Module (ULM), 6-Minute Walk Test (6MWT), compound muscle

SUBMITTER: Darras BT 

PROVIDER: S-EPMC6541434 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature