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Intramuscular injection is the recommended and safer route

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Pipeline strategies include (i) AAV gene replacement for SLC52A2/A3 , (ii) brain-targeted delivery of active flavins, and (iii) small-molecule chaperones that stabilize misfolded transportersall of which will require biomarker-guided protocols and functional imaging endpoints to quantify long-term efficacy (19)

Acknowledgements This work was supported by the Tehran University of Medical Sciences, Deputy of Research