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Published on: August 10, 2018
Administration of Onasemnogene Abeparvovec in an Infant With Spinal Muscular Atrophy and PCR-Confirmed SARS-CoV-2
Ikushi Shimomura1, Shinsuke Maruyama1, Yuichi Kodama1
1Department of Pediatrics, Graduate School of Medical and Dental Sciences, Kagoshima University, Kagoshima, JPN.
Insights
Early spinal muscular atrophy (SMA) treatment with onasemnogene abeparvovec (OA) may be safe in asymptomatic infants with SARS-CoV-2. This case study suggests timely OA administration is possible following maternal COVID-19 exposure.
Area of Science:
- Neurology
- Genetics
- Pediatrics
Background:
- Spinal muscular atrophy (SMA) requires timely treatment with onasemnogene abeparvovec (OA) for optimal motor function.
- Immune responses to OA and optimal timing post-SARS-CoV-2 infection are not well-established.
- COVID-19 exposure poses a challenge for initiating critical SMA therapy.
Abstract:
Onasemnogene abeparvovec (OA) is used to treat spinal muscular atrophy (SMA), and early treatment is critical. However, OA administration may trigger an immune response. To date, no studies have established the optimal timing for OA administration following SARS-CoV-2 infection. We report the case of an eight-month-old female infant presenting with delayed motor development who was diagnosed with SMA. OA therapy was initially prescribed but postponed because of a risk of COVID-19 exposure; the patient's mother had recently developed COVID-19. Despite testing positive for SARS-CoV-2, the infant remained asymptomatic, and her viral load did not increase. OA was administered 19 days after the mother's COVID-19 diagnosis. The patient did not develop COVID-19 and experienced no significant adverse effects. This case suggests that OA administration may be safe in asymptomatic SARS-CoV-2-positive patients with low viral loads, allowing timely therapy to preserve motor function. Further studies are needed to establish evidence-based guidelines for the optimal timing of OA administration.
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