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Page 1
Longitudinal biomarkers in amyotrophic lateral sclerosis.
Huang F, Zhu Y, Hsiao-Nakamoto J, Tang X, Dugas JC, Moscovitch-Lopatin M, Glass JD, Brown RH Jr, Ladha SS, Lacomis D, Harris JM, Scearce-Levie K, Ho C, Bowser R, Berry JD. Huang F, et al. Among authors: hsiao nakamoto j. Ann Clin Transl Neurol. 2020 Jul;7(7):1103-1116. doi: 10.1002/acn3.51078. Epub 2020 Jun 9. Ann Clin Transl Neurol. 2020. PMID: 32515902 Free PMC article.
Safety, pharmacokinetics and target engagement of novel RIPK1 inhibitor SAR443060 (DNL747) for neurodegenerative disorders: Randomized, placebo-controlled, double-blind phase I/Ib studies in healthy subjects and patients.
Vissers MFJM, Heuberger JAAC, Groeneveld GJ, Oude Nijhuis J, De Deyn PP, Hadi S, Harris J, Tsai RM, Cruz-Herranz A, Huang F, Tong V, Erickson R, Zhu Y, Scearce-Levie K, Hsiao-Nakamoto J, Tang X, Chang M, Fox BM, Estrada AA, Pomponio RJ, Alonso-Alonso M, Zilberstein M, Atassi N, Troyer MD, Ho C. Vissers MFJM, et al. Among authors: hsiao nakamoto j. Clin Transl Sci. 2022 Aug;15(8):2010-2023. doi: 10.1111/cts.13317. Epub 2022 Jun 1. Clin Transl Sci. 2022. PMID: 35649245 Free PMC article. Clinical Trial.
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Logan T, Simon MJ, Rana A, Cherf GM, Srivastava A, Davis SS, Low RLY, Chiu CL, Fang M, Huang F, Bhalla A, Llapashtica C, Prorok R, Pizzo ME, Calvert MEK, Sun EW, Hsiao-Nakamoto J, Rajendra Y, Lexa KW, Srivastava DB, van Lengerich B, Wang J, Robles-Colmenares Y, Kim DJ, Duque J, Lenser M, Earr TK, Nguyen H, Chau R, Tsogtbaatar B, Ravi R, Skuja LL, Solanoy H, Rosen HJ, Boeve BF, Boxer AL, Heuer HW, Dennis MS, Kariolis MS, Monroe KM, Przybyla L, Sanchez PE, Meisner R, Diaz D, Henne KR, Watts RJ, Henry AG, Gunasekaran K, Astarita G, Suh JH, Lewcock JW, DeVos SL, Di Paolo G. Logan T, et al. Among authors: hsiao nakamoto j. Cell. 2021 Sep 2;184(18):4651-4668.e25. doi: 10.1016/j.cell.2021.08.002. Epub 2021 Aug 26. Cell. 2021. PMID: 34450028 Free PMC article.
Decreased lipidated ApoE-receptor interactions confer protection against pathogenicity of ApoE and its lipid cargoes in lysosomes.
Guo JL, Braun D, Fitzgerald GA, Hsieh YT, Rougé L, Litvinchuk A, Steffek M, Propson NE, Heffner CM, Discenza C, Han SJ, Rana A, Skuja LL, Lin BQ, Sun EW, Davis SS, Balasundar S, Becerra I, Dugas JC, Ha C, Hsiao-Nakamoto J, Huang F, Jain S, Kung JE, Liau NPD, Mahon CS, Nguyen HN, Nguyen N, Samaddar M, Shi Y, Tatarakis D, Tian Y, Zhu Y, Suh JH, Sandmann T, Calvert MEK, Arguello A, Kane LA, Lewcock JW, Holtzman DM, Koth CM, Di Paolo G. Guo JL, et al. Among authors: hsiao nakamoto j. Cell. 2024 Nov 11:S0092-8674(24)01209-1. doi: 10.1016/j.cell.2024.10.027. Online ahead of print. Cell. 2024. PMID: 39532095 Free article.
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Logan T, Simon MJ, Rana A, Cherf GM, Srivastava A, Davis SS, Yoon Low RL, Chiu CL, Fang M, Huang F, Bhalla A, Llapashtica C, Prorok R, Pizzo ME, Calvert MEK, Sun EW, Hsiao-Nakamoto J, Rajendra Y, Lexa KW, Srivastava DB, van Lengerich B, Wang J, Robles-Colmenares Y, Kim DJ, Duque J, Lenser M, Earr TK, Nguyen H, Chau R, Tsogtbaatar B, Ravi R, Skuja LL, Solanoy H, Rosen HJ, Boeve BF, Boxer AL, Heuer HW, Dennis MS, Kariolis MS, Monroe KM, Przybyla L, Sanchez PE, Meisner R, Diaz D, Henne KR, Watts RJ, Henry AG, Gunasekaran K, Astarita G, Suh JH, Lewcock JW, DeVos SL, Di Paolo G. Logan T, et al. Among authors: hsiao nakamoto j. Cell. 2024 Mar 14;187(6):1565-1566. doi: 10.1016/j.cell.2024.02.015. Cell. 2024. PMID: 38490183 Free article. No abstract available.
Alterations in Lysosomal, Glial and Neurodegenerative Biomarkers in Patients with Sporadic and Genetic Forms of Frontotemporal Dementia.
Hsiao-Nakamoto J, Chiu CL, VandeVrede L, Ravi R, Vandenberg B, De Groot J, Tsogtbaatar B, Fang M, Auger P, Gould NS, Marchioni F, Powers CA, Davis SS, Suh JH, Alkabsh J, Heuer HW, Lago AL, Scearce-Levie K, Seeley WW, Boeve BF, Rosen HJ, Berger A, Tsai R, Di Paolo G, Boxer AL, Bhalla A, Huang F; ALLFTD Consortium. Hsiao-Nakamoto J, et al. bioRxiv [Preprint]. 2024 Feb 12:2024.02.09.579529. doi: 10.1101/2024.02.09.579529. bioRxiv. 2024. PMID: 38405775 Free PMC article. Preprint.
Safety, pharmacokinetics, and target engagement of a brain penetrant RIPK1 inhibitor, SAR443820 (DNL788), in healthy adult participants.
Hincelin-Mery A, Nicolas X, Cantalloube C, Pomponio R, Lewanczyk P, Benamor M, Ofengeim D, Krupka E, Hsiao-Nakamoto J, Eastenson A, Atassi N. Hincelin-Mery A, et al. Among authors: hsiao nakamoto j. Clin Transl Sci. 2024 Jan;17(1):e13690. doi: 10.1111/cts.13690. Epub 2023 Dec 11. Clin Transl Sci. 2024. PMID: 38010108 Free PMC article. Clinical Trial.