Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

You may be eligible if

• Inclusion
• Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene.
• Weight ≥ 8.5 kg and ≤ 21 kg at the time of Screening Visit 2
• Naive to treatment or have discontinued an approved drug/therapy
• Exclusion:
• Previous OAV101 use or previous use of any adeno-associated virus serotype 9 (AAV9) gene therapy
• BMI \< 3rd percentile
• Participant with history of aspiration pneumonia or signs of aspiration
• Elevated anti-AAV9 antibody
• History of gene therapy, hematopoietic transplantation, or solid organ transplantation
• Inability to take corticosteroids
• Concomitant use of immunosuppressive therapy
• Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation 9. Administration of vaccines 2 weeks prior to infusion of OAV101
• Awake hypoxemia or awake oxygen saturation level decrease
• Hepatic dysfunction
• Presence of a confirmed or suspected infection
• If previously treated with disease modifying therapy, specified washout times apply
• Documented any parental consanguinity.