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Structure-function relationship in serine hydroxymethyltransferase

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Clinical trials have established the proof-of-concept for intrathecal ASO therapy, exemplified by tofersen for SOD1-ALS, which achieved accelerated approval based on biomarker reduction despite not meeting the primary clinical endpoint in its phase 3 VALOR trial (8, 43, 44)

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From a strictly harm-reduction standpoint, restoring legal compounding access under physician oversight is arguably safer than maintaining a black-market status quo