Publications by authors named "Mike A Storey"
Pediatrics
September 2020
Article Synopsis
- Spinal muscular atrophy (SMA), a major cause of infant mortality, is treated with the gene therapy onasemnogene abeparvovec-xioi, which was approved by the FDA for children under 2 years old who aren't in end-stage weakness.* !
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- A study in Ohio involving 21 children aged 1-23 months showed that gene therapy was safe, with manageable side effects, particularly in those under 6 months old, and required increased medication for older children due to more frequent enzyme elevation.* !
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- Outcomes indicated that 90% of children experienced an asymptomatic drop in platelets, with significant improvements in motor function observed after treatment, suggesting early promise for this gene replacement
