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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 ( 3.2 Multiple acyl-CoA dehydrogenase deficiency Multiple acyl-CoA dehydrogenase deficiency (MADD) also known as glutaric academia type II or lipid-storage myopathyis caused by loss-of-function variants in ETFDH, ETFA, ETFB or, more rarely, FLAD1
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Frequency recommendations vary by injury severity
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However, because these drugs carry costs, side effects, and access issues, botanicals that can support endogenous GLP-1 activity are attractive as adjunctive options
doi:10.21037/atm-21-5043 161
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