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Editorial
. 2023 Jul;46(4):539-540.
doi: 10.1002/jimd.12623. Epub 2023 May 17.

Is it time to start to consider treating the liver in glutaric aciduria type 1?

Sander Houten  1 , Curtis R Coughlin 2nd  2
Affiliations
Editorial

Is it time to start to consider treating the liver in glutaric aciduria type 1?

Sander Houten et al. J Inherit Metab Dis. 2023 Jul.
No abstract available

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References

REFERENCES

    1. Funk CB, Prasad AN, Frosk P, et al. Neuropathological, biochemical and molecular findings in a glutaric acidemia type 1 cohort. Brain. 2005;128:711-722.
    1. Sauer SW, Okun JG, Fricker G, et al. Intracerebral accumulation of glutaric and 3-hydroxyglutaric acids secondary to limited flux across the blood-brain barrier constitute a biochemical risk factor for neurodegeneration in glutaryl-CoA dehydrogenase deficiency. J Neurochem. 2006;97:899-910.
    1. Barzi M, Johnson CG, Chen T, et al. Rescue of glutaric aciduria type I in mice by liver-directed therapies. Sci Transl Med. 2023;15:eadf4086.

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