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Jakkamsetti et al., 2013
Gene therapy approaches (betibeglogene autotemcel/Zynteglo, exagamglogene autotemcel/Casgevy) received FDA approval in 2023 for eligible patients 12 years
As weve seen in our own program over the last year, the benefits of these medications far outweigh the risks which is why we understand and support patients who feel like they dont want to quit the medications cold turkey. Keep in mind that the FDA has approved the medications for indefinite use
Neurons can engage additional protective strategies under extreme proteostasis disruption
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