Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Miller, Timothy, Cudkowicz, Merit, Shaw, Pamela J., Andersen, Peter M., Atassi, Nazem, Bucelli, Robert C., Genge, Angela, Glass, Jonathan, Ladha, Shafeeq, Ludolph, Albert L., Maragakis, Nicholas J., McDermott, Christopher J., Pestronk, Alan, Ravits, John, Salachas, François, Trudell, Randall, Van Damme, Philip, Zinman, Lorne, Bennett, C. Frank, Lane, Roger, Sandrock, Alfred, Runz, Heiko, Graham, Danielle, Houshyar, Hani, McCampbell, Alexander, Nestorov, Ivan, Chang, Ih, McNeill, Manjit, Fanning, Laura, Fradette, Stephanie, Ferguson, Toby A.·2020·New England Journal of Medicine
Cited by (565)
Items 101–200 of 565
- Amyotrophic Lateral Sclerosis: Proteins, Proteostasis, Prions, and PromisesLuke McAlary, Yee Lian Chew, Jeremy Stephen Lum, Nicholas J. Geraghty, Justin J. Yerbury, Neil R. Cashman · 2020 · Frontiers in Cellular Neuroscience
- APOE loss-of-function variants: Compatible with longevity and associated with resistance to Alzheimer’s disease pathologyChemparathy, Augustine, Le Guen, Yann, Chen, Sunny, Lee, Eun-Gyung, Leong, Lesley, Gorzynski, John E., Jensen, Tanner D., Ferrasse, Alexis, Xu, Guangxue, Xiang, Hong et al. · 2024 · Neuron
- Translational Approaches for Brain Delivery of Biologics via Cerebrospinal FluidShraddha Sadekar, Mayumi Bowen, Hao Cai, Samira Jamalian, Hanine Rafidi, Whitney Shatz-Binder, Julien Lafrance‐Vanasse, Pamela Chan, William J. Meilandt, Amy Oldendorp et al. · 2022 · Clinical Pharmacology & Therapeutics
- Gene Therapy in ALS and SMA: Advances, Challenges and PerspectivesJan Lejman, Kinga Panuciak, Emilia Nowicka, Angelika Mastalerczyk, Katarzyna Wojciechowska, Monika Lejman · 2023 · International Journal of Molecular Sciences
- Multiomics and machine-learning identify novel transcriptional and mutational signatures in amyotrophic lateral sclerosisAlberto Catanese, Sandeep Rajkumar, Daniel Sommer, Pegah Masrori, Nicole Hersmus, Philip Van Damme, Simon Witzel, Albert Christian Ludolph, Ritchie Ho, Tobias M. Boeckers et al. · 2023 · Brain
- Opinion: more mouse models and more translation needed for ALSElizabeth Fisher, Linda Greensmith, Andrea Malaspina, Pietro Fratta, Michael G. Hanna, Giampietro Schiavo, Adrian M. Isaacs, Richard W. Orrell, Thomas J. Cunningham, Abraham Acevedo‐Arozena · 2023 · Molecular Neurodegeneration
- Therapies in preclinical and clinical development for Angelman syndromeTheodora Markati, Jessica B. Duis, Laurent Servais · 2021 · Expert Opinion on Investigational Drugs
- Sensory Involvement in Amyotrophic Lateral SclerosisM.Á. Rubio, Mireia Herrando‐Grabulosa, Xavier Navarro · 2022 · International Journal of Molecular Sciences
- Performance of serum neurofilament light chain in a wide spectrum of clinical courses of amyotrophic lateral sclerosis—a cross‐sectional multicenter studyThomas Meyer, Erma Salkic, Torsten Grehl, Ute Weyen, Dagmar Kettemann, Patrick Weydt, René Günther, Paul Lingor, Jan Christoph Koch, Susanne Petri et al. · 2023 · European Journal of Neurology
- Antisense Oligonucleotides (ASOs) in Motor Neuron Diseases: A Road to Cure in Light and ShadeSilvia Cantara, Giorgia Simoncelli, Claudia Ricci · 2024 · International Journal of Molecular Sciences
- Overview of current and emerging therapies for amytrophic lateral sclerosisJack J. Chen, PharmD, BCPS, BCGP, FASCP, FCCP; · 2020 · The American Journal of Managed Care
- Micro-RNAs Shuttled by Extracellular Vesicles Secreted from Mesenchymal Stem Cells Dampen Astrocyte Pathological Activation and Support Neuroprotection in In-Vitro Models of ALSFrancesca Provenzano, Sophie Nyberg, Debora Giunti, Carola Torazza, Benedetta Parodi, Tiziana Bonifacino, Cesare Usai, Nicole Kerlero de Rosbo, Marco Milanese, Antonio Uccelli et al. · 2022 · Cells
- Efficacy and safety of low-dose IL-2 as an add-on therapy to riluzole (MIROCALS): a phase 2b, double-blind, randomised, placebo-controlled trialGilbert Bensimon, P. Nigel Leigh, Timothy I. M. Tree, Andrea Malaspina, Christine Payan, Hang‐Phuong Pham, Pieter Klaassen, Pamela J. Shaw, Ahmad Al Khleifat, Maria del Mar Amador et al. · 2025 · The Lancet
- Nanotechnology-enabled gene delivery for cancer and other genetic diseasesTong Jiang, Karina Marie Gonzalez, Leyla Estrella Cordova, Jianqin Lu · 2023 · Expert Opinion on Drug Delivery
- Tofersen for SOD1 ALSWilliam Everett, Robert C. Bucelli · 2024 · Neurodegenerative Disease Management
- Tofersen for SOD1 amyotrophic lateral sclerosis: a systematic review and meta-analysisAbdullah Ashraf Hamad, Ibraheem M. Alkhawaldeh, Abdulqadir J. Nashwan, Mostafa Mahmoud Meshref, Yahia Zakaria Bashier Imam · 2025 · Neurological Sciences
- The Inflammatory Puzzle: Piecing together the Links between Neuroinflammation and Amyotrophic Lateral SclerosisDi He, Yan Chun Xu, Mingsheng Liu, Liying Cui · 2023 · Aging and Disease
- Unraveling the Genetic Landscape of Neurological Disorders: Insights into Pathogenesis, Techniques for Variant Identification, and Therapeutic ApproachesZeba Firdaus, Xiaogang Li · 2024 · International Journal of Molecular Sciences
- Frontotemporal Pathology in Motor Neuron Disease Phenotypes: Insights From NeuroimagingMary Clare McKenna, Philippe Corcia, Philippe Couratier, We Fong Siah, Pierre‐François Pradat, Peter Bede · 2021 · Frontiers in Neurology
- Amyotrophic Lateral Sclerosis Clinical Trials and Interpretation of Functional End Points and Fluid BiomarkersJeremy M. Shefner, Richard Bedlack, Jinsy A. Andrews, James D. Berry, Robert P. Bowser, Robert H. Brown, Jonathan D. Glass, Nicholas John Maragakis, Timothy M. Miller, Jeffrey D. Rothstein et al. · 2022 · JAMA Neurology
- Early activation of cellular stress and death pathways caused by cytoplasmic TDP-43 in the rNLS8 mouse model of ALS and FTDWei Luan, Amanda L. Wright, Heledd Brown-Wright, Sheng Le, Rebecca San Gil, Lidia Madrid San Martin, Karen Ling, Paymaan Jafar‐Nejad, Frank Rigo, Adam K. Walker · 2023 · Molecular Psychiatry
- Serious Neurologic Adverse Events in Tofersen Clinical Trials for Amyotrophic Lateral SclerosisAlexandra Lovett, Sowmya Chary, Suma Babu, Gaëlle Bruneteau, Jonathan D. Glass, Merete Karlsborg, Shafeeq S. Ladha, Keith Mayl, Christopher J. McDermott, Robert C. Bucelli et al. · 2025 · Muscle & Nerve
- Beyond the Traditional Clinical Trials for Amyotrophic Lateral Sclerosis and The Future Impact of Gene TherapyM Cappella, Pierre‐François Pradat, Giorgia Querin, Maria Grazia Biferi · 2020 · Journal of Neuromuscular Diseases
- Ibudilast (MN-166) in amyotrophic lateral sclerosis- an open label, safety and pharmacodynamic trialSuma Babu, Baileigh G. Hightower, James Gin Man Chan, Nicole R. Zürcher, Pia Kivisäkk, Chieh-En Jane Tseng, Danica L. Sanders, Ashley Robichaud, Haruhiko Banno, Armineuza Evora et al. · 2021 · NeuroImage Clinical
- A Phase 1 study of GDC ‐0134, a dual leucine zipper kinase inhibitor, in ALSJonathan Katz, Jeffrey D. Rothstein, Merit Cudkowicz, Angela L. Genge, Björn Oskarsson, Avis Brennan Hains, Chen Chen, Joshua Galanter, Braydon L. Burgess, Yong‐Yeon Cho et al. · 2022 · Annals of Clinical and Translational Neurology
- Biomarkers for amyotrophic lateral sclerosisSimon Witzel, Kristina Mayer, Patrick Oeckl · 2022 · Current Opinion in Neurology
- Approaches to gene modulation therapy for ALSMeijboom, Katharina E., Brown, Robert H. · 2022 · Neurotherapeutics
- Cerebrospinal Fluid Neurofilament Light Chain (NfL) Predicts Disease Aggressiveness in Amyotrophic Lateral Sclerosis: An Application of the D50 Disease Progression ModelMarie Dreger, Robert Steinbach, Nayana Gaur, Klara Metzner, Beatrice Stubendorff, Otto Wilhelm Witte, Julian Großkreutz · 2021 · Frontiers in Neuroscience
- Amyotrophe Lateralsklerose (ALS) – Diagnose, Verlauf und neue BehandlungsoptionenThomas Meyer · 2021 · DMW - Deutsche Medizinische Wochenschrift
- Astrocytes in Neurodegeneration: Inspiration From GeneticsJingxuan Huang, Chunyu Li, Huifang Shang · 2022 · Frontiers in Neuroscience
- Between hope and reality: treatment of genetic diseases through nucleic acid-based drugsVirginie Baylot, Thi Khanh Le, David Taïeb, Palma Rocchi, Laurence Colleaux · 2024 · Communications Biology
- Downregulating PTBP1 Fails to Convert Astrocytes into Hippocampal Neurons and to Alleviate Symptoms in Alzheimer's Mouse ModelsTiantian Guo, Xinjia Pan, Guangtong Jiang, Denghong Zhang, Jinghui Qi, Lin Shao, Zhanxiang Wang, Huaxi Xu, Yingjun Zhao · 2022 · Journal of Neuroscience
- The Role of Sphingomyelin and Ceramide in Motor Neuron DiseasesGavin McCluskey, Colette G. Donaghy, Karen E. Morrison, John McConville, William John Duddy, Stéphanie Duguez · 2022 · Journal of Personalized Medicine
- Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injectionByrnes, Amy E., Dominguez, Sara L., Yen, Chun-Wan, Laufer, Benjamin I., Foreman, Oded, Reichelt, Mike, Lin, Han, Sagolla, Meredith, Hötzel, Kathy, Ngu, Hai et al. · 2023 · Molecular Therapy - Nucleic Acids
- Genetic epidemiology of familial ALS in BrazilJoão Pedro Nunes Gonçalves, Tauana Bernardes Leoni, Melina P. Martins, Thiago Mazzo Peluzzo, Mário Emílio Teixeira Dourado Júnior, Jonas Alex Morales Saute, Anna Paula Paranhos Miranda Covaleski, Acary Souza Bullé Oliveira, Rinaldo Claudino, Wilson Marques et al. · 2021 · Neurobiology of Aging
- Current Concepts on Genetic Aspects of Mitochondrial Dysfunction in Amyotrophic Lateral SclerosisJankovic, Milena, Novakovic, Ivana, Gamil Anwar Dawod, Phepy, Gamil Anwar Dawod, Ayman, Drinic, Aleksandra, Abdel Motaleb, Fayda I., Ducic, Sinisa, Nikolic, Dejan · 2021 · International Journal of Molecular Sciences
- ASO drug Qalsody (tofersen) targets amyotrophic lateral sclerosisJing Jin, Xiao‐bo Zhong · 2023 · Trends in Pharmacological Sciences
- Clinical and patient‐reported outcomes and neurofilament response during tofersen treatment in SOD1 ‐related ALS —A multicenter observational study over 18 monthsThomas Meyer, Peggy Schumann, Patrick Weydt, Susanne Petri, Jochen Hans Weishaupt, Ute Weyen, Jan Christoph Koch, René Günther, Martin Regensburger, Matthias Boentert et al. · 2024 · Muscle & Nerve
- Lipid nanoparticles and transcranial focused ultrasound enhance the delivery of SOD1 antisense oligonucleotides to the murine brain for ALS therapyGayathri R. Ediriweera, Amal J. Sivaram, Gary Cowin, Mikayla L. Brown, Luke McAlary, Jeremy Stephen Lum, Nicholas L. Fletcher, Liam Robinson, Joshua D. Simpson, Liyu Chen et al. · 2024 · Journal of Controlled Release
- Intrathecal Therapies for Neurodegenerative Diseases: A Review of Current Approaches and the Urgent Need for Advanced Delivery SystemsThomas Gabriel Schreiner, Manuel Menéndez‐González, Oliver Daniel Schreiner, Romeo Cristian Ciobanu · 2025 · Biomedicines
- Cortical progression patterns in individual ALS patients across multiple timepoints: a mosaic-based approach for clinical useMarlene Tahedl, Rangariroyashe H. Chipika, Jasmin Lope, Stacey Li Hi Shing, Orla M. Hardiman, Peter Bede · 2021 · Journal of Neurology
- Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a “real‐world” settingSam Smith, Kelly McCoy‐Gross, Amber Malcolm, Jeri Oranski, Jesse Markway, Timothy M. Miller, Robert C. Bucelli · 2025 · Annals of Clinical and Translational Neurology
- Defective Oligodendroglial Lineage and Demyelination in Amyotrophic Lateral SclerosisÉlisabeth Traiffort, Séverine Morisset‐Lopez, Mireille Moussaed, Amina Zahaf · 2021 · International Journal of Molecular Sciences
- Breached Barriers: A Scoping Review of Blood-Central Nervous System Barrier Pathology in Amyotrophic Lateral SclerosisArio Mirian, Alexander Joseph Moszczynski, Serena Soleimani, Isabelle Aubert, Lorne H. Zinman, Agessandro Abrahão · 2022 · Frontiers in Cellular Neuroscience
- Biochemical and clinical biomarkers in adult SMA 3–4 patients treated with nusinersen for 22 monthsBram De Wel, Maxim De Schaepdryver, Koen Poesen, Kristl G. Claeys · 2022 · Annals of Clinical and Translational Neurology
- Phosphorylated neurofilament heavy chain: a potential diagnostic biomarker in amyotrophic lateral sclerosisIlana Heckler, Iswariya Venkataraman · 2022 · Journal of Neurophysiology
- Effect of RNS60 in amyotrophic lateral sclerosis: a phase II multicentre, randomized, double‐blind, placebo‐controlled trialEttore Beghi, Elisabetta Pupillo, Elisa Bianchi, Valentina Bonetto, Silvia Luotti, Laura Pasetto, Caterina Bendotti, Massimo Tortarolo, Francesca M. Sironi, Laura Camporeale et al. · 2022 · European Journal of Neurology
- Presymptomatic grey matter alterations in ALS kindreds: a computational neuroimaging study of asymptomatic C9orf72 and SOD1 mutation carriersPeter Bede, Dorothée E. Lulé, Hans‐Peter Müller, Ee Ling Tan, Johannes Dorst, Albert Christian Ludolph, Jan Kassubek · 2023 · Journal of Neurology
- LncRNA EILA promotes CDK4/6 inhibitor resistance in breast cancer by stabilizing cyclin E1 proteinZijie Cai, Qianfeng Shi, Yudong Li, Liang Jin, Shunying Li, Lok Lam Wong, Jingru Wang, Xiaoting Jiang, Mengdi Zhu, Jinna Lin et al. · 2023 · Science Advances
- Neurofilament Biomarkers in Neurology: From Neuroinflammation to Neurodegeneration, Bridging Established and Novel Analytical Advances with Clinical PracticeAriadne Daponte, Christos Koros, Charalampos Skarlis, Daphne Siozios, Michail Rentzos, Sokratis G. Papageorgiou, Maria C. Anagnostouli · 2025 · International Journal of Molecular Sciences
- Intrathecal delivery of AAV-NDNF ameliorates disease progression of ALS miceWei Cheng, Jing Huang, Xiu-Qing Fu, Wei-Ya Tian, Peng-Ming Zeng, Yang Li, Zhen‐Ge Luo · 2023 · Molecular Therapy
- Alteration of Mitochondrial Integrity as Upstream Event in the Pathophysiology of SOD1-ALSRené Günther, Arun Pal, Chloe Williams, Vitaly L. Zimyanin, Maria Liehr, Cläre von Neubeck, Mechthild Krause, Mrudula G. Parab, Susanne Petri, Norman Kalmbach et al. · 2022 · Cells
- Value-Generating Exploratory Trials in Neurodegenerative DementiasLauren G. Friedman, Nicholas McKeehan, Yuko Hara, Jeffrey L. Cummings, Dawn C. Matthews, Jian Zhu, Richard C. Mohs, Deli Wang, Suzanne B. Hendrix, Melanie Quintana et al. · 2021 · Neurology
- Neuroinflammation in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia and the Interest of Induced Pluripotent Stem Cells to Study Immune Cells Interactions With NeuronsElise Liu, Léa Karpf, Delphine Bohl · 2021 · Frontiers in Molecular Neuroscience
- A copper chaperone–mimetic polytherapy for SOD1-associated amyotrophic lateral sclerosisLuke McAlary, Victoria K. Shephard, Gareth S. A. Wright, Justin J. Yerbury · 2022 · Journal of Biological Chemistry
- In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse modelYi A. Chen, Mark W. Kankel, Sam Hana, Shukkwan Kelly Lau, Maria I. Zavodszky, Olivia P. McKissick, Nicole Mastrangelo, Jessica Dion, Bin Wang, Daniel Ferretti et al. · 2022 · Gene Therapy
- The MOE Modification of RNA: Origins and Widescale Impact on the Oligonucleotide Therapeutics FieldAlyssa C. Hill, Jonathan Hall · 2023 · Helvetica Chimica Acta
- Therapeutic targeting of ALS pathways: Refocusing an incomplete pictureNicholas John Maragakis, Mamede A. de Carvalho, Michael D. Weiss · 2023 · Annals of Clinical and Translational Neurology
- Advances and Challenges in Gene Therapy for Neurodegenerative Diseases: A Systematic ReviewNerea García-González, Jaime Gonçalves-Sánchez, Ricardo Gómez‐Nieto, Jesús María Gonçalves, Dolores E. López · 2024 · International Journal of Molecular Sciences
- Parkinson’s disease therapy: what lies ahead?Andreas Wolfgang Wolff, Nicolas U Schumacher, Dominik Pürner, Gerrit Machetanz, Antonia Franziska Demleitner, Emily Feneberg, Maike Hagemeier, Paul Lingor · 2023 · Journal of Neural Transmission
- Astrocytes: Dissecting Their Diverse Roles in Amyotrophic Lateral Sclerosis and Frontotemporal DementiaValori, Chiara F., Sulmona, Claudia, Brambilla, Liliana, Rossi, Daniela · 2023 · Cells
- Long-term treatment of SOD1 ALS with tofersen: a multicentre experience in 17 patientsMario Sabatelli, Federica Cerri, Riccardo Zuccarino, Agata Katia Patanella, Daniela Bernardo, Giulia Bisogni, Raffaella Tanel, Valeria Sansone, Massimiliano Filosto, Serena Lattante et al. · 2024 · Journal of Neurology
- Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulationRachael Miller, Joseph D. Paquette, Alexandra Barker, Ellen Sapp, Nicholas McHugh, Brianna M. Bramato, Nozomi Yamada, Julia F. Alterman, Dimas Echeverria, Ken Yamada et al. · 2024 · Molecular Therapy — Nucleic Acids
- The blood biomarkers puzzle – A review of protein biomarkers in neurodegenerative diseasesChristian Barro, Henrik Zetterberg · 2021 · Journal of Neuroscience Methods
- Urinary neopterin: A novel biomarker of disease progression in amyotrophic lateral sclerosisStephanie R. Shepheard, Vassilios Karnaros, Beben Benyamin, David W Schultz, Megan Fowler, Joanne Wuu, Tim Chataway, Andrea Malaspina, Michael G Benatar, Mary‐Louise Rogers · 2021 · European Journal of Neurology
- Status of ALS treatment, insights into therapeutic challenges and dilemmasKhamaysa, Mohammed, Pradat, Pierre-François · 2022 · Journal of Personalized Medicine
- Antisense therapies in neurological diseasesJ.-B. Brunet de Courssou, Alexandra Dürr, David Adams, Jean‐Christophe Corvol, Louise‐Laure Mariani · 2021 · Brain
- SYF2 suppression mitigates neurodegeneration in models of diverse forms of ALSGabriel R. Linares, Yichen Li, Wen-Hsuan Chang, Jasper Rubin-Sigler, Stacee Mendonca, Sarah Hong, Yunsun Eoh, Wenxuan Guo, Yi‐Hsuan Huang, Jonathan Chang et al. · 2023 · Cell stem cell
- Amyotrophic lateral sclerosisDavid S. Younger, Robert H. Brown · 2023 · Handbook of clinical neurology
- Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selectionShukkwan K. Chen, Zachary C. E. Hawley, Maria I. Zavodszky, Sam Hana, Daniel Ferretti, Branka Grubor, Michael C. Hawes, Shanqin Xu, Stefan Hamann, Galina Marsh et al. · 2023 · Molecular Therapy — Nucleic Acids
- The new direction of drug development: Degradation of undruggable targets through targeting chimera technologyXuewu Liang, Hairu Ren, Fengyang Han, Renwen Liang, Jiayan Zhao, Hong Liu · 2023 · Medicinal Research Reviews
- Activating autophagy to eliminate toxic protein aggregates with small molecules in neurodegenerative diseasesYuqi Fu, Jin Zhang, Rui Qin, Yueting Ren, Tingting Zhou, Bo Han, Bo Liu · 2025 · Pharmacological Reviews
- Blood β-Synuclein and Neurofilament Light Chain During the Course of Prion DiseaseSteffen Halbgebauer, Samir Abu‐Rumeileh, Patrick Oeckl, Petra Steinacker, Francesco Roselli, Diana Wiesner, Angela Mammana, Michael Beekes, Izaro Kortazar Zubizarreta, Guiomar Pérez de Nanclares et al. · 2022 · Neurology
- Prionoids in amyotrophic lateral sclerosisPhilippe Gosset, William Camu, Cédric Raoul, Alexandre Mezghrani · 2022 · Brain Communications
- Intercellular transmission of pathogenic proteins in ALS: Exploring the pathogenic waveFrederick J. Arnold, Ashlie Nguyen, Richard Bedlack, Craig L. Bennett, Albert R. La Spada · 2023 · Neurobiology of Disease
- Metabolic Transporters in the Peripheral Nerve—What, Where, and Why?Atul Kumar Rawat, Brett M. Morrison · 2021 · Neurotherapeutics
- Regional variability and genotypic and pharmacodynamic effects on PrP concentration in the CNSMeredith A Mortberg, Hien Tran Zhao, Andrew G. Reidenbach, Juliana E Gentile, Eric W. Kuhn, Jill O’Moore, Patrick M. Dooley, Theresa R. Connors, Curt Mazur, Shona W. Allen et al. · 2022 · JCI Insight
- Potential of Cellular Therapy for ALS: Current Strategies and Future ProspectsTing-Jung Lin, Kuang-Chao Cheng, Luo-Yun Wu, Wei-Yu Lai, Thai‐Yen Ling, Yung‐Che Kuo, Yen‐Hua Huang · 2022 · Frontiers in Cell and Developmental Biology
- Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In VitroLoren L. Flynn, Ruohan Li, Ianthe L. Pitout, May Thandar Aung-Htut, Leon M. Larcher, Jack A. L. Cooper, K. Greer, Alysia Hubbard, Lisa Griffiths, Charles S. Bond et al. · 2022 · Frontiers in Genetics
- Clinical and genetic features of amyotrophic lateral sclerosis patients withC9orf72mutationsMaximilian Wiesenfarth, Kornelia Günther, Kathrin Müller, Simon Witzel, Ulrike Weiland, Kristina Mayer, Christine H. Herrmann, Dávid Brenner, Joachim Schuster, Axel Freischmidt et al. · 2023 · Brain Communications
- Limited value of serum neurofilament light chain in diagnosing amyotrophic lateral sclerosisJennifer C. Davies, Thanuja Dharmadasa, Alexander G. Thompson, Evan Edmond, Katie Tharshana Yoganathan, Jiali Gao, Kevin Talbot, Martin R. Turner · 2023 · Brain Communications
- Cerebrospinal Fluid and Blood Neurofilament Light Chain Protein in Prion Disease and Other Rapidly Progressive Dementias: Current State of the ArtSamir Abu‐Rumeileh, Piero Parchi · 2021 · Frontiers in Neuroscience
- Consideration of C9orf72-associated ALS-FTD as a neurodevelopmental disorder: insights from neuroimagingPeter Bede, We Fong Siah, Mary Clare McKenna, Stacey Li Hi Shing · 2020 · Journal of Neurology Neurosurgery & Psychiatry
- Targeting for Success: Demonstrating Proof-of-Concept with Mechanistic Early Phase Clinical Pharmacology Studies for Disease-Modification in Neurodegenerative DisordersMaurits F. J. M. Vissers, Jules A. A. C. Heuberger, Geert Jan Groeneveld · 2021 · International Journal of Molecular Sciences
- Why TDP-43? Why Not? Mechanisms of Metabolic Dysfunction in Amyotrophic Lateral SclerosisMara-Luciana Floare, Scott P. Allen · 2020 · Neuroscience Insights
- Advances in the genetic classification of amyotrophic lateral sclerosisJohnathan Cooper‐Knock, Calum Harvey, Sai Zhang, Tobias Moll, Ilia Sarah Timpanaro, Kevin Patrick Kenna, Alfredo Iacoangeli, Jan Herman Veldink · 2021 · Current Opinion in Neurology
- Updates on Disease Mechanisms and Therapeutics for Amyotrophic Lateral SclerosisLien Nguyen · 2024 · Cells
- Sodium Phenylbutyrate and Tauroursodeoxycholic Acid: A Story of Hope Turned to Disappointment in Amyotrophic Lateral Sclerosis TreatmentArsh Haj Mohamad Ebrahim Ketabforoush, Faezeh Faghihi, Fereshteh Azedi, Armin Ariaei, Mohamad Amin Habibi, Maryam Khalili, Bahram Haghi Ashtiani, Mohammad Taghi Joghataei, William David Arnold · 2024 · Clinical Drug Investigation
- Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy PatientsLars Hendrik Müschen, Alma Osmanovic, Camilla Binz, Konstantin Fritz Jendretzky, Gresa Ranxha, Paul Bronzlik, Omar Abu-Fares, Flavia Wiehler, Nora Möhn, Martin Werner Hümmert et al. · 2021 · Brain Sciences
- From bench side to clinic: Potential and challenges of RNA vaccines and therapeutics in infectious diseasesPandey, Manish, Ojha, Divya, Bansal, Sakshi, Rode, Ambadas B., Chawla, Geetanjali · 2021 · Molecular Aspects of Medicine
- Synucleinopathy in Amyotrophic Lateral Sclerosis: A Potential Avenue for Antisense Therapeutics?Bradley Roberts, Frances Theunissen, Francis L. Mastaglia, Patrick Anthony Akkari, Loren L. Flynn · 2022 · International Journal of Molecular Sciences
- Prospects for gene replacement therapies in amyotrophic lateral sclerosisIlaria Giovannelli, Adrian Higginbottom, Janine Kirby, Mimoun Azzouz, Pamela J. Shaw · 2022 · Nature Reviews Neurology
- Intrathecal administration of a novel siRNA modality extends survival and improves motor function in the SOD1G93A ALS mouse modelChunling Duan, Moorim Kang, Xiaojie Pan, Zubao Gan, Vera Huang, Guanlin Li, Robert F. Place, Long‐Cheng Li · 2024 · Molecular Therapy — Nucleic Acids
- CSF neurofilament light chain profiling and quantitation in neurological diseasesClaire Alexandra Leckey, John B. Coulton, Tatiana A. Giovannucci, Yingxin He, Aram Aslanyan, Rhiannon Laban, Amanda Heslegrave, Ivan Doykov, Francesca Ammoscato, Jeremy Chataway et al. · 2024 · Brain Communications
- Recent Progress of Antisense Oligonucleotide Therapy for Superoxide-Dismutase-1-Mutated Amyotrophic Lateral Sclerosis: Focus on TofersenHidenori Moriyama, Toshifumi Yokota · 2024 · Genes
- Gene therapy breakthroughs in ALS: a beacon of hope for 20% of ALS patientsQingjian Xie, Kezheng Li, Yinuo Chen, Yaojia Li, Wenhua Jiang, Wen Cao, Huan Yu, Dongsheng Fan, Binbin Deng · 2025 · Translational Neurodegeneration
- FGF23, a novel muscle biomarker detected in the early stages of ALSYing Si, Mohamed Kazamel, Michael G Benatar, Joanne Wuu, Yuri Kwon, Thaddaeus Kwan, Nan Jiang, Dominik Kentrup, Christian Faul, Lyndsy Alesce et al. · 2021 · Scientific Reports
- Amyotrophic lateral sclerosis with SOD1 mutations shows distinct brain metabolic changesAntonio Canosa, Andrea Calvo, Cristina Moglia, Rosario Vasta, Francesca Palumbo, Luca Solero, Francesca Di Pede, Sara Cabras, Vincenzo Arena, Grazia Zocco et al. · 2022 · European Journal of Nuclear Medicine and Molecular Imaging
- The landscape of cognitive impairment in superoxide dismutase 1-amyotrophic lateral sclerosisIlaria Martinelli, Elisabetta Zucchi, Cecilia Simonini, Giulia Gianferrari, Giovanna Zamboni, Marcello Pinti, Jessica Mandrioli · 2022 · Neural Regeneration Research
- Palliative care principles in ALSChristen L. Shoesmith · 2023 · Handbook of clinical neurology