Results from a phase 1 study of nusinersen (ISIS-SMN Rx ) in children with spinal muscular atrophy
The University of Texas Southwestern Medical Center · Boston Children's Hospital · +1 more institution
Abstract
Objective: To examine safety, tolerability, pharmacokinetics, and preliminary clinical efficacy of intrathecal nusinersen (previously ISIS-SMN Rx ), an antisense oligonucleotide designed to alter splicing of SMN2 mRNA, in patients with childhood spinal muscular atrophy (SMA).
Citation impact
- FWCI
- 54.42
- Percentile
- 100%
- References
- 27
Authors
9- CAClaudia A. ChiribogaCorresponding
The University of Texas Southwestern Medical Center, Boston Children's Hospital, University of Utah
- KJKathryn J. Swoboda
University of Utah, Boston Children's Hospital, The University of Texas Southwestern Medical Center
- BTBasil T. Darras
Boston Children's Hospital, University of Utah, The University of Texas Southwestern Medical Center
- STSusan T. Iannaccone
The University of Texas Southwestern Medical Center, Boston Children's Hospital, University of Utah
- JMJacqueline Montes
University of Utah, Boston Children's Hospital, The University of Texas Southwestern Medical Center
Topics & keywords
- Tolerability
- Spinal muscular atrophy
- SMA*
- Medicine
- Pharmacokinetics
- Adverse effect
- Cohort
- Anesthesia