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Prospective characterization of early symptom onset and progression in young pediatric patients with variants in the G LA gene across 5 years: Longitudinal data from the Fabry MOPPet Study.
Laney DA, Houde MF, Foley AL, Peck DS, Atherton AM, Manwaring LP, Grange DK, Heese BA, Holida MD, Quillin AL, Vinson R, Auray-Blais C, Hopkin RJ. Laney DA, et al. Among authors: auray blais c. Genet Med Open. 2024 Sep 10;2:101891. doi: 10.1016/j.gimo.2024.101891. eCollection 2024. Genet Med Open. 2024. PMID: 39669636 Free PMC article.
A case of hyperlysinemia identified by urine newborn screening.
Yeganeh M, Auray-Blais C, Maranda B, Sabovic A, DeVita RJ, Lazarus MB, Houten SM. Yeganeh M, et al. Among authors: auray blais c. JIMD Rep. 2023 Oct 22;64(6):440-445. doi: 10.1002/jmd2.12399. eCollection 2023 Nov. JIMD Rep. 2023. PMID: 37927488 Free PMC article.
Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease.
Saleh AH, Rothe M, Barber DL, McKillop WM, Fraser G, Morel CF, Schambach A, Auray-Blais C, West ML, Khan A, Fowler DH, Rupar CA, Foley R, Medin JA, Keating A. Saleh AH, et al. Among authors: auray blais c. Mol Ther Methods Clin Dev. 2023 Jan 18;28:262-271. doi: 10.1016/j.omtm.2023.01.003. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36816757 Free PMC article.
111 results