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Editorial
. 2021 May;44(3):515-517.
doi: 10.1002/jimd.12377. Epub 2021 Mar 25.

Regulatory news: Dojolvi (triheptanoin) as a source of calories and fatty acids in long-chain fatty acid oxidation disorders: FDA approval summary

Dina Zand  1 , Jenny Doan  1 , Sojeong Yi  2 , Jie Wang  2 , Lian Ma  2 , Emmanuel Akinshola  1 , Sushanta Chakder  1 , Joette Meyer  1 , Michael Pacanowski  2 , Laura Lee Johnson  3 , Kathleen Donohue  1 , Julie Beitz  1
Affiliations
Editorial

Regulatory news: Dojolvi (triheptanoin) as a source of calories and fatty acids in long-chain fatty acid oxidation disorders: FDA approval summary

Dina Zand et al. J Inherit Metab Dis. 2021 May.
No abstract available

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References

REFERENCES

    1. Merritt JL 2nd, Norris M, Kanungo S. Fatty acid oxidation disorders. Ann Transl Med. 2018;6(24):473.
    1. Saudubray JM, van den Berghe G, Walter JH, eds. Inborn Metabolic Diseases: Diagnosis and Treatment. 5th ed. Heidelberg, Germany: Springer-Verlag Berlin Heidelberg; 2012:205.
    1. Engel WK, Vick NA, Glueck CJ, Levy RI. A skeletal-muscle disorder associated with intermittent symptoms and a possible defect of lipid metabolism. N Engl J Med. 1970;282(13):697-704.
    1. Gillingham MB, Heitner SB, Martin J, et al. Triheptanoin vs trioctanoin for long-chain fatty acid oxidation disorders: a double blinded, randomized controlled trial. J Inherit Metab Dis. 2017;40(6):831-843.
    1. Vockley J, Burton B, Berry GT, et al. Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fatty acid oxidation disorders (LC-FAOD). J Inherit Metab Dis. 2019;42(1):169-177.

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