Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study

Authors: van den Berg, Leonard H ^*, Rothstein, Jeffrey D, Shaw, Pamela J, Babu, Suma, Benatar, Michael, Bucelli, Robert C, Genge, Angela, Glass, Jonathan D, Hardiman, Orla, Libri, Vincenzo, Mobach, Theodore, Oskarsson, Björn, Pattee, Gary L, Ravits, John, Shaw, Christopher E, Weber, Markus, Zinman, Lorne, Jafar-nejad, Paymaan, Rigo, Frank, Lin, Luan, Ferguson, Toby A, Gotter, Anthony L, Graham, Danielle, Monine, Michael, Inra, Jennifer, Sinks, Susie, Eraly, Satish, Garafalo, Steve, Fradette, Stephanie

Source: In The Lancet Neurology September 2024 23(9):901-912

Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study

Authors: Benatar, Michael, Wuu, Joanne, Andersen, Peter M., Bucelli, Robert C., Andrews, Jinsy A., Otto, Markus, Farahany, Nita A., Harrington, Elizabeth A., Chen, Weiping, Mitchell, Adele A., Ferguson, Toby, Chew, Sheena, Gedney, Liz, Oakley, Sue, Heo, Jeong, Chary, Sowmya, Fanning, Laura, Graham, Danielle, Sun, Peng, Liu, Yingying, Wong, Janice, Fradette, Stephanie

Source: In Neurotherapeutics July 2022 19(4):1248-1258

Autologistic network model on binary data for disease progression study

Authors: Shin, Yei Eun, Sang, Huiyan, Liu, Dawei, Ferguson, Toby A., Song, Peter X. K.

Source: Biometrics, 2019 Dec 01. 75(4), 1310-1320.

Access URL: https://www.jstor.org/stable/45238772

Implementing Motor Unit Number Index (MUNIX) in a large clinical trial: Real world experience from 27 centres

Authors: Neuwirth, Christoph, Braun, Nathalie, Claeys, Kristl G., Bucelli, Robert, Fournier, Christina, Bromberg, Mark, Petri, Susanne, Goedee, Stephan, Lenglet, Timothée, Leppanen, Ron, Canosa, Antonio, Goodman, Ira, Al-Lozi, Muhammad, Ohkubo, Takuya, Hübers, Annemarie, Atassi, Nazem, Abrahao, Agessandro, Funke, Andreas, Appelfeller, Martin, Tümmler, Anke, Finegan, Eoin, Glass, Jonathan D., Babu, Suma, Ladha, Shafeeq S., Kwast-Rabben, Olga, Juntas-Morales, Raul, Coffey, Amina, Chaudhry, Vinay, Vu, Tuan, Saephanh, Chow, Newhard, Colleen, Zakrzewski, Marion, Rosier, Esther, Hamel, Nancy, Raheja, Divisha, Raaijman, Jesper, Ferguson, Toby, Weber, Markus

Source: In Clinical Neurophysiology August 2018 129(8):1756-1762

Increasing the efficiency of clinical trials in neurodegenerative disorders using group sequential trial designs

Authors: van Eijk, Ruben P.A., Nikolakopoulos, Stavros, Ferguson, Toby A., Liu, Dawei, Eijkemans, Marinus J.C., van den Berg, Leonard H.

Source: In Journal of Clinical Epidemiology June 2018 98:80-88

Palmitoylation controls DLK localization, interactions and activity to ensure effective axonal injury signaling

Authors: Holland, Sabrina M., Collura, Kaitlin M., Ketschek, Andrea, Noma, Kentaro, Ferguson, Toby A., Jin, Yishi, Gallo, Gianluca, Thomas, Gareth M.

Source: Proceedings of the National Academy of Sciences of the United States of America, 2016 Jan . 113(3), 763-768.

Access URL: https://www.jstor.org/stable/26467460

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Calpains mediate axonal cytoskeleton disintegration during Wallerian degeneration

Authors: Ma, Marek, Ferguson, Toby A., Schoch, Kathleen M., Li, Jian, Qian, Yaping, Shofer, Frances S., Saatman, Kathryn E., Neumar, Robert W.

Source: In Neurobiology of Disease August 2013 56:34-46

Missense Mutations in the Copper Transporter Gene ATP7A Cause X-Linked Distal Hereditary Motor Neuropathy

Authors: Kennerson, Marina L., Nicholson, Garth A., Kaler, Stephen G., Kowalski, Bartosz, Mercer, Julian F.B., Tang, Jingrong, Llanos, Roxana M., Chu, Shannon, Takata, Reinaldo I., Speck-Martins, Carlos E., Baets, Jonathan, Almeida-Souza, Leonardo, Fischer, Dirk, Timmerman, Vincent, Taylor, Philip E., Scherer, Steven S., Ferguson, Toby A., Bird, Thomas D., De Jonghe, Peter, Feely, Shawna M.E., Shy, Michael E., Garbern, James Y.

Source: In The American Journal of Human Genetics 2010 86(3):343-352

Increased Risk of Venous Thromboembolism in Patients With Amyotrophic Lateral Sclerosis: Results From a US Insurance Claims Database Study.

Authors: Kupelian, Varant, Viscidi, Emma, Hall, Susan, Li, Li, Eaton, Susan, Dilley, Anne, Currier, Nicolas, Ferguson, Toby, Fanning, Laura

Source: Neurology: Clinical Practice; Feb2023, Vol. 13 Issue 1, p1-8, 8p

A pediatric quantitative systems pharmacology model of neurofilament trafficking in spinal muscular atrophy treated with the antisense oligonucleotide nusinersen.

Authors: Paris, Alessio, Bora, Pranami, Parolo, Silvia, MacCannell, Drew, Monine, Michael, van der Munnik, Nick, Tong, Xiao, Eraly, Satish, Berger, Zdenek, Graham, Danielle, Ferguson, Toby, Domenici, Enrico, Nestorov, Ivan, Marchetti, Luca

Source: CPT: Pharmacometrics & Systems Pharmacology; Feb2023, Vol. 12 Issue 2, p196-206, 11p

Subject Terms: SPINAL muscular atrophy, CYTOPLASMIC filaments, TREATMENT effectiveness, PHARMACOLOGY, THERAPEUTICS

An age‐dependent mathematical model of neurofilament trafficking in healthy conditions.

Authors: Paris, Alessio, Bora, Pranami, Parolo, Silvia, Monine, Michael, Tong, Xiao, Eraly, Satish, Masson, Eric, Ferguson, Toby, McCampbell, Alexander, Graham, Danielle, Domenici, Enrico, Nestorov, Ivan, Marchetti, Luca

Source: CPT: Pharmacometrics & Systems Pharmacology; Apr2022, Vol. 11 Issue 4, p447-457, 11p

Subject Terms: MATHEMATICAL models, CYTOPLASMIC filaments, ORDINARY differential equations, NERVOUS system, NEURODEGENERATION, MOTOR neuron diseases, AMPA receptors, CEREBROSPINAL fluid

Location identification in a communications signalling network

Location identification in a communications signalling network

Correction to: Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study

Authors: Benatar, Michael, Wuu, Joanne, Andersen, Peter M., Bucelli, Robert C., Andrews, Jinsy A., Otto, Markus, Farahany, Nita A., Harrington, Elizabeth A., Chen, Weiping, Mitchell, Adele A., Ferguson, Toby, Chew, Sheena, Gedney, Liz, Oakley, Sue, Heo, Jeong, Chary, Sowmya, Fanning, Laura, Graham, Danielle, Sun, Peng, Liu, Yingying, Wong, Janice, Fradette, Stephanie

Source: In Neurotherapeutics September 2022 19(5):1686-1686

Regeneration of Axons after Nerve Transection Repair Is Enhanced by Degradation of Chondroitin Sulfate Proteoglycan

Authors: Zuo, Jian, Neubauer, Debbie, Graham, James, Krekoski, Craig A., Ferguson, Toby A., Muir, David

Source: In Experimental Neurology July 2002 176(1):221-228

Gold Coast diagnostic criteria: Implications for ALS diagnosis and clinical trial enrollment.

Authors: Vucic, Steve¹ (AUTHOR) steve.vucic@sydney.edu.au, Ferguson, Toby A.² (AUTHOR), Cummings, Catherine³ (AUTHOR), Hotchkin, Michael T.⁴ (AUTHOR), Genge, Angela⁵ (AUTHOR), Glanzman, Robert⁴ (AUTHOR), Roet, Kasper C. D.⁶ (AUTHOR), Cudkowicz, Merit⁷ (AUTHOR), Kiernan, Matthew C.⁸ (AUTHOR)

Source: Muscle & Nerve. Nov2021, Vol. 64 Issue 5, p532-537. 6p.

MMP-2 and MMP-9 Increase the Neurite-Promoting Potential of Schwann Cell Basal Laminae and Are Upregulated in Degenerated Nerve

Authors: Ferguson, Toby A., Muir, David

Source: In Molecular and Cellular Neuroscience August 2000 16(2):157-167

Clinical presentation and diagnosis of Amyotrophic Lateral Sclerosis.

Authors: Ferguson, Toby A.¹, Elman, Lauren B.¹ elmanl@uphs.upenn.edu

Source: NeuroRehabilitation. 2007, Vol. 22 Issue 6, p409-416. 8p. 1 Diagram, 4 Charts.

Subject Terms: *AMYOTROPHIC lateral sclerosis, *MOTOR neuron diseases, *NEUROMUSCULAR diseases, *DEGLUTITION disorders, *NERVOUS system

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