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Page 1
Optimizing human α-galactosidase for treatment of Fabry disease.
Hallows WC, Skvorak K, Agard N, Kruse N, Zhang X, Zhu Y, Botham RC, Chng C, Shukla C, Lao J, Miller M, Sero A, Viduya J, Ismaili MHA, McCluskie K, Schiffmann R, Silverman AP, Shen JS, Huisman GW. Hallows WC, et al. Among authors: mccluskie k. Sci Rep. 2023 Mar 23;13(1):4748. doi: 10.1038/s41598-023-31777-4. Sci Rep. 2023. PMID: 36959353 Free PMC article.
Oral enzyme therapy for maple syrup urine disease (MSUD) suppresses plasma leucine levels in intermediate MSUD mice and healthy nonhuman primates.
Skvorak K, Liu J, Kruse N, Mehmood R, Das S, Jenne S, Chng C, Lao UL, Duan D, Asfaha J, Du F, Teadt L, Sero A, Ching C, Riggins J, Pope L, Yan P, Mashiana H, Ismaili MHA, McCluskie K, Huisman G, Silverman AP. Skvorak K, et al. Among authors: mccluskie k. J Inherit Metab Dis. 2023 Nov;46(6):1089-1103. doi: 10.1002/jimd.12662. Epub 2023 Aug 9. J Inherit Metab Dis. 2023. PMID: 37494004
An orally administered enzyme therapeutic for homocystinuria that suppresses homocysteine by metabolizing methionine in the gastrointestinal tract.
Skvorak K, Mitchell V, Teadt L, Franklin KA, Lee HO, Kruse N, Huitt-Roehl C, Hang J, Du F, Galanie S, Guan S, Aijaz H, Zhang N, Rajkovic G, Kruger WD, Ismaili MHA, Huisman G, McCluskie K, Silverman AP. Skvorak K, et al. Among authors: mccluskie k. Mol Genet Metab. 2023 Aug;139(4):107653. doi: 10.1016/j.ymgme.2023.107653. Epub 2023 Jul 12. Mol Genet Metab. 2023. PMID: 37463544 Free article.
23 results