Works by Miller, Timothy M.

Results: 65
    1

    Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid–siRNA conjugate administered into cerebrospinal fluid Open Access.

    Published in:
    Nucleic Acids Research, 2025, v. 53, n. 12, p. 1, doi. 10.1093/nar/gkaf600
    By:
    • Sorets, Alexander G;
    • Schwensen, Katrina R;
    • Francini, Nora;
    • Kjar, Andrew;
    • Abdulrahman, Adam M;
    • Shostak, Alena;
    • Katdare, Ketaki A;
    • Schoch, Kathleen M;
    • Cowell, Rebecca P;
    • Park, Joshua C;
    • Ligocki, Alexander P;
    • Ford, William T;
    • Ventura-Antunes, Lissa;
    • Hoogenboezem, Ella N;
    • Prusky, Alex;
    • Castleberry, Mark;
    • Michell, Danielle L;
    • Fritsch, Emma;
    • Lyons, Sarah M;
    • Miller, Timothy M
    Publication type:
    Article
    2

    Health utilities and quality-adjusted life years for patients with amyotrophic lateral sclerosis receiving reldesemtiv or placebo in FORTITUDE-ALS.

    Published in:
    Journal of Medical Economics, 2023, v. 26, n. 1, p. 488, doi. 10.1080/13696998.2023.2192588
    By:
    • Gebrehiwet, Paulos;
    • Meng, Lisa;
    • Rudnicki, Stacy A.;
    • Sarocco, Phil;
    • Wei, Jenny;
    • Wolff, Andrew A.;
    • Butzner, Michael;
    • Chiò, Adriano;
    • Andrews, Jinsy A.;
    • Genge, Angela;
    • Hughes, Dyfrig A.;
    • Jackson, Carlayne E.;
    • Lechtzin, Noah;
    • Miller, Timothy M.;
    • Shefner, Jeremy M.
    Publication type:
    Article
    3

    MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models.

    Published in:
    Journal of Neuroscience, 2017, v. 37, n. 22, p. 5574, doi. 10.1523/JNEUROSCI.3582-16.2017
    By:
    • Hoye, Mariah L.;
    • Koval, Erica D.;
    • Wegener, Amy J.;
    • Hyman, Theodore S.;
    • Chengran Yang;
    • O'Brien, David R.;
    • Miller, Rebecca L.;
    • Cole, Tracy;
    • Schoch, Kathleen M.;
    • Tao Shen;
    • Tomonori Kunikata;
    • Richard, Jean-Philippe;
    • Gutmann, David H.;
    • Maragakis, Nicholas J.;
    • Kordasiewicz, Holly B.;
    • Dougherty, Joseph D.;
    • Miller, Timothy M.
    Publication type:
    Article
    4
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    Reldesemtiv in Amyotrophic Lateral Sclerosis: Results From the COURAGE-ALS Randomized Clinical Trial.

    Published in:
    JAMA Neurology, 2025, v. 82, n. 5, p. 477, doi. 10.1001/jamaneurol.2025.0241
    By:
    • Shefner, Jeremy M.;
    • Cudkowicz, Merit E.;
    • Genge, Angela;
    • Hardiman, Orla;
    • Al-Chalabi, Ammar;
    • Andrews, Jinsy A.;
    • Chio, Adriano;
    • Corcia, Philippe;
    • Couratier, Philippe;
    • de Carvalho, Mamede;
    • Heiman-Patterson, Terry;
    • Henderson, Robert D.;
    • Ingre, Caroline;
    • Johnston, Wendy;
    • Ludolph, Albert;
    • Maragakis, Nicholas J.;
    • Miller, Timothy M.;
    • Mora, Jesus S.;
    • Petri, Susanne;
    • Simmons, Zachary
    Publication type:
    Article
    6
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    TREM2 Variant p.R47H as a Risk Factor for Sporadic Amyotrophic Lateral Sclerosis.

    Published in:
    JAMA Neurology, 2014, v. 71, n. 4, p. 449, doi. 10.1001/jamaneurol.2013.6237
    By:
    • Cady, Janet;
    • Koval, Erica D.;
    • Benitez, Bruno A.;
    • Zaidman, Craig;
    • Jockel-Balsarotti, Jennifer;
    • Allred, Peggy;
    • Baloh, Robert H.;
    • Ravits, John;
    • Simpson, Ericka;
    • Appel, Stanley H.;
    • Pestronk, Alan;
    • Goate, Alison M.;
    • Miller, Timothy M.;
    • Cruchaga, Carlos;
    • Harms, Matthew B.
    Publication type:
    Article
    8

    Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD.

    Published in:
    Nature Communications, 2022, v. 13, n. 1, p. 1, doi. 10.1038/s41467-022-30387-4
    By:
    • Krishnan, Gopinath;
    • Raitcheva, Denitza;
    • Bartlett, Daniel;
    • Prudencio, Mercedes;
    • McKenna-Yasek, Diane M.;
    • Douthwright, Catherine;
    • Oskarsson, Björn E.;
    • Ladha, Shafeeq;
    • King, Oliver D.;
    • Barmada, Sami J.;
    • Miller, Timothy M.;
    • Bowser, Robert;
    • Watts, Jonathan K.;
    • Petrucelli, Leonard;
    • Brown, Robert H.;
    • Kankel, Mark W.;
    • Gao, Fen-Biao
    Publication type:
    Article
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    In vivo kinetic approach reveals slow SOD1 turnover in the CNS.

    Published in:
    Journal of Clinical Investigation, 2015, v. 125, n. 7, p. 2772, doi. 10.1172/JCI80705
    By:
    • Crisp, Matthew J.;
    • Mawuenyega, Kwasi C.;
    • Patterson, Bruce W.;
    • Reddy, Naveen C.;
    • Chott, Robert;
    • Self, Wade K.;
    • Weihl, Conrad C.;
    • Jockel-Balsarotti, Jennifer;
    • Varadhachary, Arun S.;
    • Bucelli, Robert C.;
    • Yarasheski, Kevin E.;
    • Bateman, Randall;
    • Miller, Timothy M.
    Publication type:
    Article
    12

    Antisense oligonucleotide therapy for neurodegenerative disease.

    Published in:
    2006
    By:
    • Smith, Richard A.;
    • Miller, Timothy M.;
    • Yamanaka, Koji;
    • Monia, Brett P.;
    • Condon, Thomas P.;
    • Hung, Gene;
    • Lobsiger, Christian S.;
    • Ward, Chris M.;
    • McAlonis-Downes, Melissa;
    • Wei, Hongbing;
    • Wancewicz, Ed V.;
    • Bennett, C. Frank;
    • Cleveland, Don W.
    Publication type:
    journal article
    13
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    Fibroblast bioenergetics to classify amyotrophic lateral sclerosis patients.

    Published in:
    Molecular Neurodegeneration, 2017, v. 12, p. 1, doi. 10.1186/s13024-017-0217-5
    By:
    • Konrad, Csaba;
    • Hibiki Kawamata;
    • Bredvik, Kirsten G.;
    • Arreguin, Andrea J.;
    • Cajamarca, Steven A.;
    • Hupf, Jonathan C.;
    • Ravits, John M.;
    • Miller, Timothy M.;
    • Maragakis, Nicholas J.;
    • Hales, Chadwick M.;
    • Glass, Jonathan D.;
    • Gross, Steven;
    • Hiroshi Mitsumoto;
    • Manfredi, Giovanni
    Publication type:
    Article
    15

    Access for ALL in ALS: A large‐scale, inclusive, collaborative consortium to unlock the molecular and genetic mechanisms of amyotrophic lateral sclerosis.

    Published in:
    Muscle & Nerve, 2024, v. 70, n. 6, p. 1140, doi. 10.1002/mus.28244
    By:
    • Berry, James D.;
    • Paganoni, Sabrina;
    • Harms, Matthew B.;
    • Shneider, Neil;
    • Andrews, Jinsy;
    • Miller, Timothy M.;
    • Babu, Suma;
    • Sherman, Alex V.;
    • Harris, Brent T.;
    • Provenzano, Frank A.;
    • Phatnani, Hemali P.;
    • Shefner, Jeremy;
    • Garret, Mark A.;
    • Ladha, Shaffeeq S.;
    • Tsou, Amy Y.;
    • Mohan, Praveena;
    • Igne, Courtney;
    • Bowser, Robert
    Publication type:
    Article
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    Reply.

    Published in:
    2015
    By:
    • Paganoni, Sabrina;
    • Schoenfeld, David;
    • Shui, Amy;
    • Cudkowicz, Merit;
    • Miller, Timothy M.
    Publication type:
    Letter
    22
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    COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success.

    Published in:
    Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration, 2023, v. 24, n. 5/6, p. 523, doi. 10.1080/21678421.2023.2216223
    By:
    • Shefner, Jeremy M.;
    • Al-Chalabi, Ammar;
    • Andrews, Jinsy A.;
    • Chio, Adriano;
    • De Carvalho, Mamede;
    • Cockroft, Bettina M.;
    • Corcia, Philippe;
    • Couratier, Philippe;
    • Cudkowicz, Merit E.;
    • Genge, Angela;
    • Hardiman, Orla;
    • Heiman-Patterson, Terry;
    • Henderson, Robert D.;
    • Ingre, Caroline;
    • Jackson, Carlayne E.;
    • Johnston, Wendy;
    • Lechtzin, Noah;
    • Ludolph, Albert;
    • Maragakis, Nicholas J.;
    • Miller, Timothy M.
    Publication type:
    Article
    25
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    Prescription and acceptance of durable medical equipment in FORTITUDE-ALS, a study of reldesemtiv in ALS: post hoc analyses of a randomized, double-blind, placebo-controlled clinical trial.

    Published in:
    Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration, 2022, v. 23, n. 3/4, p. 263, doi. 10.1080/21678421.2021.1946083
    By:
    • Rudnicki, Stacy A.;
    • Andrews, Jinsy A.;
    • Genge, Angela;
    • Jackson, Carlayne;
    • Lechtzin, Noah;
    • Miller, Timothy M.;
    • Cockroft, Bettina M.;
    • Malik, Fady I.;
    • Meng, Lisa;
    • Wei, Jenny;
    • Wolff, Andrew A.;
    • Shefner, Jeremy M.
    Publication type:
    Article
    27

    A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS.

    Published in:
    Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration, 2021, v. 22, n. 3/4, p. 287, doi. 10.1080/21678421.2020.1822410
    By:
    • Shefner, Jeremy M.;
    • Andrews, Jinsy A.;
    • Genge, Angela;
    • Jackson, Carlayne;
    • Lechtzin, Noah;
    • Miller, Timothy M.;
    • Cockroft, Bettina M.;
    • Meng, Lisa;
    • Wei, Jenny;
    • Wolff, Andrew A.;
    • Malik, Fady I.;
    • Bodkin, Cynthia;
    • Brooks, Benjamin R.;
    • Caress, James;
    • Dionne, Annie;
    • Fee, Dominic;
    • Goutman, Stephen A.;
    • Goyal, Namita A.;
    • Hardiman, Orla;
    • Hayat, Ghazala
    Publication type:
    Article
    28

    Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy.

    Published in:
    Science Translational Medicine, 2017, v. 9, n. 374, p. 1, doi. 10.1126/scitranslmed.aag0481
    By:
    • DeVos, Sarah L.;
    • Miller, Rebecca L.;
    • Schoch, Kathleen M.;
    • Holmes, Brandon B.;
    • Kebodeaux, Carey S.;
    • Wegener, Amy J.;
    • Guo Chen;
    • Tao Shen;
    • Tran, Hien;
    • Nichols, Brandon;
    • Zanardi, Tom A.;
    • Kordasiewicz, Holly B.;
    • Swayze, Eric E.;
    • Bennett, C. Frank;
    • Diamond, Marc I.;
    • Miller, Timothy M.
    Publication type:
    Article
    29

    Antisense Reduction of Tau in Adult Mice Protects against Seizures.

    Published in:
    Journal of Neuroscience, 2013, v. 33, n. 31, p. 12887, doi. 10.1523/JNEUROSCI.2107-13.2013
    By:
    • DeVos, Sarah L.;
    • Goncharoff, Dustin K.;
    • Chen, Guo;
    • Kebodeaux, Carey S.;
    • Yamada, Kaoru;
    • Stewart, Floy R.;
    • Schuler, Dorothy R.;
    • Maloney, Susan E.;
    • Wozniak, David F.;
    • Rigo, Frank;
    • Bennett, C. Frank;
    • Cirrito, John R.;
    • Holtzman, David M.;
    • Miller, Timothy M.
    Publication type:
    Article
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    Has gene therapy for ALS arrived?

    Published in:
    Nature Medicine, 2003, v. 9, n. 10, p. 1256, doi. 10.1038/nm1003-1256
    By:
    • Miller, Timothy M.;
    • Cleveland, Don W.
    Publication type:
    Article
    34
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    A Path to High-Efficiency Optical Coupling for HIRMES.

    Published in:
    Journal of Low Temperature Physics, 2018, v. 193, n. 5/6, p. 681, doi. 10.1007/s10909-018-1939-7
    By:
    • Miller, Timothy M.;
    • Brown, Ari-David;
    • Costen, Nicholas;
    • Franz, David;
    • Kutyrev, Alexander;
    • Mikula, Vilem;
    • Miller, Kevin H.;
    • Moseley, S. Harvey;
    • Oxborrow, Joseph;
    • Rostem, Karwan;
    • Wollack, Edward J.
    Publication type:
    Article
    36
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    Network analysis of the cerebrospinal fluid proteome reveals shared and unique differences between sporadic and familial forms of amyotrophic lateral sclerosis.

    Published in:
    Molecular Neurodegeneration, 2025, v. 20, n. 1, p. 1, doi. 10.1186/s13024-025-00838-9
    By:
    • Trautwig, Adam N.;
    • Fox, Edward J.;
    • Dammer, Eric B.;
    • Shantaraman, Anantharaman;
    • Ping, Lingyan;
    • Duong, Duc M.;
    • Watson, Caroline M.;
    • Wu, Fang;
    • Asress, Seneshaw;
    • Guo, Qi;
    • Levey, Allan I.;
    • Lah, James J.;
    • Verde, Federico;
    • Doretti, Alberto;
    • Ratti, Antonia;
    • Ticozzi, Nicola;
    • Ly, Cindy V.;
    • Miller, Timothy M.;
    • Garret, Mark A.;
    • Berry, James D.
    Publication type:
    Article
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    Protein kinetics of superoxide dismutase‐1 in familial and sporadic amyotrophic lateral sclerosis.

    Published in:
    Annals of Clinical & Translational Neurology, 2023, v. 10, n. 6, p. 1012, doi. 10.1002/acn3.51784
    By:
    • Ly, Cindy V.;
    • Ireland, Margaret D.;
    • Self, Wade K.;
    • Bollinger, James;
    • Jockel‐Balsarotti, Jennifer;
    • Herzog, Hillary;
    • Allred, Peggy;
    • Miller, Leah;
    • Doyle, Michael;
    • Anez‐Bruzual, Isabel;
    • Trikamji, Bhavesh;
    • Hyman, Ted;
    • Kung, Tyler;
    • Nicholson, Katherine;
    • Bucelli, Robert C.;
    • Patterson, Bruce W.;
    • Bateman, Randall J.;
    • Miller, Timothy M.
    Publication type:
    Article
    45

    Protein production is an early biomarker for RNA‐targeted therapies.

    Published in:
    Annals of Clinical & Translational Neurology, 2018, v. 5, n. 12, p. 1492, doi. 10.1002/acn3.657
    By:
    • Self, Wade K.;
    • Schoch, Kathleen M.;
    • Alex, Jacob;
    • Barthélemy, Nicolas;
    • Bollinger, James G.;
    • Sato, Chihiro;
    • Cole, Tracy;
    • Kordasiewicz, Holly B.;
    • Swayze, Eric;
    • Bateman, Randall J.;
    • Miller, Timothy M.
    Publication type:
    Article
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    Plasma neurofilament light predicts mortality in patients with stroke.

    Published in:
    Science Translational Medicine, 2020, v. 12, n. 569, p. 1, doi. 10.1126/scitranslmed.aay1913
    By:
    • Gendron, Tania F.;
    • Badi, Mohammed K.;
    • Heckman, Michael G.;
    • Jansen-West, Karen R.;
    • Vilanilam, George K.;
    • Johnson, Patrick W.;
    • Burch, Alexander R.;
    • Walton, Ronald L.;
    • Ross, Owen A.;
    • Brott, Thomas G.;
    • Miller, Timothy M.;
    • Berry, James D.;
    • Nicholson, Katharine A.;
    • Wszolek, Zbigniew K.;
    • Oskarsson, Björn E.;
    • Sheth, Kevin N.;
    • Sansing, Lauren H.;
    • Falcone, Guido J.;
    • Cucchiara, Brett L.;
    • Meschia, James F.
    Publication type:
    Article
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