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80 results

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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. 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.
DNL104, a Centrally Penetrant RIPK1 Inhibitor, Inhibits RIP1 Kinase Phosphorylation in a Randomized Phase I Ascending Dose Study in Healthy Volunteers.
Grievink HW, Heuberger JAAC, Huang F, Chaudhary R, Birkhoff WAJ, Tonn GR, Mosesova S, Erickson R, Moerland M, Haddick PCG, Scearce-Levie K, Ho C, Groeneveld GJ. Grievink HW, et al. Clin Pharmacol Ther. 2020 Feb;107(2):406-414. doi: 10.1002/cpt.1615. Epub 2019 Sep 25. Clin Pharmacol Ther. 2020. PMID: 31437302 Clinical Trial.
Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.
Arguello A, Meisner R, Thomsen ER, Nguyen HN, Ravi R, Simms J, Lo I, Speckart J, Holtzman J, Gill TM, Chan D, Cheng Y, Chiu CL, Dugas JC, Fang M, Lopez IA, Solanoy H, Tsogtbaatar B, Zhu Y, Bhalla A, Henne KR, Henry AG, Delucchi A, Costanzo S, Harris JM, Diaz D, Scearce-Levie K, Sanchez PE. Arguello A, et al. Among authors: scearce levie k. JCI Insight. 2021 Oct 8;6(19):e145445. doi: 10.1172/jci.insight.145445. JCI Insight. 2021. PMID: 34622797 Free PMC article.
[No title available]
[No authors listed] [No authors listed] PMID: 35109044
Molecular architecture determines brain delivery of a transferrin receptor-targeted lysosomal enzyme.
Arguello A, Mahon CS, Calvert MEK, Chan D, Dugas JC, Pizzo ME, Thomsen ER, Chau R, Damo LA, Duque J, Fang M, Giese T, Kim DJ, Liang N, Nguyen HN, Solanoy H, Tsogtbaatar B, Ullman JC, Wang J, Dennis MS, Diaz D, Gunasekaran K, Henne KR, Lewcock JW, Sanchez PE, Troyer MD, Harris JM, Scearce-Levie K, Shan L, Watts RJ, Thorne RG, Henry AG, Kariolis MS. Arguello A, et al. Among authors: scearce levie k. J Exp Med. 2022 Mar 7;219(3):e20211057. doi: 10.1084/jem.20211057. Epub 2022 Feb 28. J Exp Med. 2022. PMID: 35226042 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: scearce levie k. 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.
Preclinical and clinical evaluation of the LRRK2 inhibitor DNL201 for Parkinson's disease.
Jennings D, Huntwork-Rodriguez S, Henry AG, Sasaki JC, Meisner R, Diaz D, Solanoy H, Wang X, Negrou E, Bondar VV, Ghosh R, Maloney MT, Propson NE, Zhu Y, Maciuca RD, Harris L, Kay A, LeWitt P, King TA, Kern D, Ellenbogen A, Goodman I, Siderowf A, Aldred J, Omidvar O, Masoud ST, Davis SS, Arguello A, Estrada AA, de Vicente J, Sweeney ZK, Astarita G, Borin MT, Wong BK, Wong H, Nguyen H, Scearce-Levie K, Ho C, Troyer MD. Jennings D, et al. Among authors: scearce levie k. Sci Transl Med. 2022 Jun 8;14(648):eabj2658. doi: 10.1126/scitranslmed.abj2658. Epub 2022 Jun 8. Sci Transl Med. 2022. PMID: 35675433
LRRK2 Inhibition by BIIB122 in Healthy Participants and Patients with Parkinson's Disease.
Jennings D, Huntwork-Rodriguez S, Vissers MFJM, Daryani VM, Diaz D, Goo MS, Chen JJ, Maciuca R, Fraser K, Mabrouk OS, van de Wetering de Rooij J, Heuberger JAAC, Groeneveld GJ, Borin MT, Cruz-Herranz A, Graham D, Scearce-Levie K, De Vicente J, Henry AG, Chin P, Ho C, Troyer MD. Jennings D, et al. Among authors: scearce levie k. Mov Disord. 2023 Mar;38(3):386-398. doi: 10.1002/mds.29297. Epub 2023 Feb 18. Mov Disord. 2023. PMID: 36807624 Clinical Trial.
80 results