Jennifer Hsiao‐Nakamoto

Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic

Todd Logan, Matthew Simon, Gerald Maxwell Cherf et al. · Cell · 2021 · 210 citations · Full text

Neurodegenerative Diseases, Molecular Neuroscience, Lysosomal Storage Disorder +11

Longitudinal biomarkers in amyotrophic lateral sclerosis

Fen Huang, Yuda Zhu, Jennifer Hsiao‐Nakamoto et al. · Annals of Clinical and Translational Neurology · 2020 · 108 citations · Full text

Safety, pharmacokinetics and target engagement of novel <scp>RIPK1</scp> inhibitor <scp>SAR443060</scp> (<scp>DNL747</scp>) for neurodegenerative disorders: Randomized, <scp>placebo‐controlled</scp>, <scp>double‐blind</scp> phase I/Ib studies in healthy subjects and patients

Maurits F. J. M. Vissers, Jules A. A. C. Heuberger, Geert Jan Groeneveld et al. · Clinical and Translational Science · 2022 · 84 citations · Full text

Decreased lipidated ApoE-receptor interactions confer protection against pathogenicity of ApoE and its lipid cargoes in lysosomes

Jing Guo, Dylan Braun, Gabriel A. Fitzgerald et al. · Cell · 2024 · 63 citations · Full text

Safety, pharmacokinetics, and target engagement of a brain penetrant RIPK1 inhibitor, SAR443820 (DNL788), in healthy adult participants

Agnès Hincelin-Méry, X. Nicolas, Cathy Cantalloube et al. · Clinical and Translational Science · 2023 · 28 citations · Full text