Gene replacement therapy for spinal muscular atrophy: safety and preliminary efficacy in a Brazilian cohort
Article in Gene Therapy (June 2024)
The most recent citing publications are shown below. View all 46 publications that cite this research output on Dimensions.
Biomarkers in Gene Therapy Development for Rare Diseases
Book chapter (May 2024)
Nusinersen therapy changed the natural course of spinal muscular atrophy type 1: What about spine and hip?
Article in Journal of Children's Orthopaedics (March 2024)