Results from a phase 1 study of nusinersen (ISIS-SMN <sub>Rx</sub> ) in children with spinal muscular atrophy
Claudia A. Chiriboga(The University of Texas Southwestern Medical Center), Kathryn J. Swoboda(University of Utah), Basil T. Darras(Boston Children's Hospital), Susan T. Iannaccone(The University of Texas Southwestern Medical Center), Jacqueline Montes(University of Utah), Darryl C. De Vivo(University of Utah), Daniel A. Norris(University of Utah), C. Frank Bennett(The University of Texas Southwestern Medical Center), Kathie M. Bishop(Boston Children's Hospital)
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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).
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