Gene Replacement Therapy Fosters Age-appropriate Development in Spinal Muscular Atrophy

State of SMA Report Confirms Large Treatment-Related Improvements in Spinal Muscular Atrophy Outcomes

Gene Replacement Therapy Found to be Safe and Effective in Older Patients with Spinal Muscular Atrophy

Gene Replacement Therapy for Spinal Muscular Atrophy Associated with Normal Development

Gene Replacement Therapy for SMA is Effective in Both Pre-symptomatic Infants and Older Symptomatic Children

STR1VE-EU: Gene Replacement Therapy in Symptomatic Spinal Muscular Atrophy Is Associated with Substantial Benefit