An open-label, dose-escalation, phase 1/2A trial (STRONG; NCT03381729) evaluating safety and efficacy after intrathecal delivery of AVXS-101 (also called OAV-101 in intrathecal administration) as a treatment for SMA with 2 copies of SMN2 and deletion of SMN1 in 51 participants aged 6–60 months is ongoing and has recently (August 2021) been allowed by the FDA to resume after nearly a 2 year suspension due to safety concerns. The gene discussed is SMN1; the disease is proximal spinal muscular atrophy.