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Page 1
Current and Future Treatments for Classic Galactosemia.
J Pers Med. 2021 Jan 28;11(2):75. doi: 10.3390/jpm11020075.
J Pers Med. 2021.
PMID: 33525536
Free PMC article.
Review.
Novel mRNA therapy restores GALT protein and enzyme activity in a zebrafish model of classic galactosemia.
Delnoy B, Haskovic M, Vanoevelen J, Steinbusch LKM, Vos EN, Knoops K, Zimmermann LJI, Noga M, Lefeber DJ, Martini PGV, Coelho AI, Rubio-Gozalbo ME.
Delnoy B, et al.
J Inherit Metab Dis. 2022 Jul;45(4):748-758. doi: 10.1002/jimd.12512. Epub 2022 May 27.
J Inherit Metab Dis. 2022.
PMID: 35527402
Free PMC article.
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