GM1 Gangliosidosis-A Mini-Review.
Nicoli ER, Annunziata I, d'Azzo A, Platt FM, Tifft CJ, Stepien KM.
Front Genet. 2021 Sep 3;12:734878. doi: 10.3389/fgene.2021.734878. eCollection 2021.
Gene therapy approaches for GM1 gangliosidosis: Focus on animal and cellular studies.
Hosseini K, Fallahi J, Tabei SMB, Razban V.
Cell Biochem Funct. 2023 Dec;41(8):1093-1105. doi: 10.1002/cbf.3887. Epub 2023 Nov 29.
GM1-Gangliosidosis Type III Associated Parkinsonism.
Kaiyrzhanov R, Guliyeva U, Gulieva S, Salayev K, Mursalova A, Allahyarova P, Ferla MP, Houlden H.
Mov Disord Clin Pract. 2021 Sep 3;8(Suppl 1):S21-S23. doi: 10.1002/mdc3.13289. eCollection 2021 Aug.
GM1 gangliosidosis type II: Results of a 10-year prospective study.
D'Souza P, Farmer C, Johnston JM, Han ST, Adams D, Hartman AL, Zein W, Huryn LA, Solomon B, King K, Jordan CP, Myles J, Nicoli ER, Rothermel CE, Mojica Algarin Y, Huang R, Quimby R, Zainab M, Bowden S, Crowell A, Buckley A, Brewer C, Regier DS, Brooks BP, Acosta MT, Baker EH, Vézina G, Thurm A, Tifft CJ.
Genet Med. 2024 Jul;26(7):101144. doi: 10.1016/j.gim.2024.101144. Epub 2024 Apr 16.
Late-infantile GM1 gangliosidosis: A case report.
Noh ES, Park HM, Kim MS, Park HD, Cho SY, Jin DK.
Medicine (Baltimore). 2022 Jan 7;101(1):e28435. doi: 10.1097/MD.0000000000028435.
The natural history of Type 1 infantile GM1 gangliosidosis: A literature-based meta-analysis.
Lang FM, Korner P, Harnett M, Karunakara A, Tifft CJ.
Mol Genet Metab. 2020 Mar;129(3):228-235. doi: 10.1016/j.ymgme.2019.12.012. Epub 2019 Dec 30.
GM1-gangliosidosis: The caregivers' assessments of symptom impact and most important symptoms to treat.
Bingaman A, Waggoner C, Andrews SM, Pangonis D, Trad M, Giugliani R, Giorgino R, Jarnes J, Vakili R, Ballard V, Peay HL.
Am J Med Genet A. 2023 Feb;191(2):408-423. doi: 10.1002/ajmg.a.63038. Epub 2022 Dec 21.
Evaluation of the Landscape of Pharmacodynamic Biomarkers in GM1 and GM2 Gangliosidosis.
Stern S, Crisamore K, Li RJ, Pacanowski M, Schuck R.
Clin Transl Sci. 2025 Mar;18(3):e70176. doi: 10.1111/cts.70176.
Sialidase NEU3 action on GM1 ganglioside is neuroprotective in GM1 gangliosidosis.
Allende ML, Lee YT, Byrnes C, Li C, Tuymetova G, Bakir JY, Nicoli ER, James VK, Brodbelt JS, Tifft CJ, Proia RL.
J Lipid Res. 2023 Dec;64(12):100463. doi: 10.1016/j.jlr.2023.100463. Epub 2023 Oct 21.
GM1 gangliosidosis: review of clinical, molecular, and therapeutic aspects.
Brunetti-Pierri N, Scaglia F.
Mol Genet Metab. 2008 Aug;94(4):391-396. doi: 10.1016/j.ymgme.2008.04.012. Epub 2008 Jun 3.
