If medically appropriate, Dr
Some may also include some sort of behavior-management coaching and other potential treatment options, like nutrition counseling and/or an exercise program
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
Aunque algunos sujetos informaron efectos secundarios como nuseas u otros problemas digestivos, no hubo problemas graves
Concentrations are reported in pmol/mL of plasma
10.3390/molecules28052097 41 JeongC