An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials.

ABSTRACT:

Background

Treatment with nusinersen has demonstrated significant and clinically meaningful benefits in clinical trials in infants and children with spinal muscular atrophy (SMA).

Objective

The objective of this analysis was to characterize the safety of nusinersen across the clinical trial program in infants and children with symptomatic SMA.

Methods

An integrated safety analysis evaluated end of study data from seven completed clinical trials that enrolled infants and children with symptomatic SMA who were treated with intrathecal nusinersen or underwent sham procedures. Two of the studies were conducted in symptomatic infants with infantile-onset SMA (most likely to develop SMA type I or II) and the remaining five in symptomatic children and adolescents with later-

SUBMITTER: Darras BT 

PROVIDER: S-EPMC6776494 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature