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There is currently no specific regimen for 3-methylglutaric aciduria, and management primarily focuses on symptomatic supportive therapy, application of anti-seizure medications and assisted ventilation therapy

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(3) Jmjd3, critical for microglial cell phenotype expression, when inhibited, leads to overactivation of pro-inflammatory microglial responses, exacerbating neuroinflammation and neuronal cell death (178)

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A summary of this discussion and the Clinician Expert Workgroups' recommendations are posted publicly on the Cost Measures Information page here:

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