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Long-Term Tofersen in <i>SOD1</i> Amyotrophic Lateral Sclerosis

20261 citationsJournal Articlehybrid Open Access

Authors

Timothy M. Miller · Valve (United States)

Merit Cudkowicz · Harvard University

Pamela Shaw · Valve (United States)

Angela Genge · Valve (United States)

Gen Sobue · Aichi Medical University

Robert C. Bucelli · Valve (United States)

Adriano Chiò ·

Abstract

ClinicalTrials.gov Identifier: VALOR NCT02623699; OLE NCT03070119.

Topics & Keywords

Amyotrophic Lateral Sclerosis Research Genetic Neurodegenerative Diseases Neurogenetic and Muscular Disorders Research

UN Sustainable Development Goals

Zero hunger

Publication Details

Published in: JAMA Neurology

Volume 83, Issue 2, pp. 115-115

DOI: 10.1001/jamaneurol.2025.4946

Field-Weighted Citation Impact: 37.17

Long-Term Tofersen in <i>SOD1</i> Amyotrophic Lateral Sclerosis | Researchclopedia

Valve (United States)

Philip Van Damme · Allen Institute for Brain Science

Albert C. Ludolph · Valve (United States)

Jonathan D. Glass · Valve (United States)

Jinsy Andrews · Valve (United States)

Suma Babu · Valve (United States)

Michael Benatar · Valve (United States)

C. McDermott · Valve (United States)

François Salachas · Valve (United States)

Gaëlle Bruneteau · Valve (United States)

Ammar Al-Chalabi · Valve (United States)

Matthew Amorin · Biogen (United States)

Ivan Nestorov · Biogen (United States)

Danielle Graham · Biogen (United States)

Luan Lin · Biogen (United States)

Peng Sun · Biogen (United States)

Manjit McNeill · Biogen (United Kingdom)

Sohail Malek · Biogen (United States)

Jennifer Inra · Biogen (United States)

Steve Garafalo · Biogen (United States)

Stephanie Fradette · Biogen (United States)

VALOR and OLE Working Group

Lien Beullens · Valve (United States)

Kristl Claeys · Valve (United States)

Thomas Claeys · Valve (United States)

Goedele Couwelier · Valve (United States)

Ann D'Hondt · Valve (United States)

Elisa Debien · Valve (United States)

Sebastiaan De Keersmaecker · Valve (United States)

Laetitia della Faille · Valve (United States)

Koen Delmotte · Valve (United States)

Sofie DR Depoortere · Valve (United States)

Laura de Velder · Valve (United States)

Laurens Dobbels · Valve (United States)

Jeroen Gijs · Valve (United States)

Simon Horckmans · Valve (United States)

Nikita Lamaire · Valve (United States)

Hannelore Liessens · Valve (United States)

Pegah Masrori · Valve (United States)

Céline Nysten · Valve (United States)

Caroline Schotte · Valve (United States)

Anouk Serrien · Valve (United States)

Bart Swinnen · Valve (United States)

Petra Tilkin · Valve (United States)

Sien Hilde Van Daele · Valve (United States)

Van Damme Pa · Allen Institute for Brain Science

Jan Vynckier · Valve (United States)

Anke Wouters · Valve (United States)

Agessandro Abrahão · Valve (United States)

Mark Angle · Valve (United States)

Mohamed Badawy · Valve (United States)

Maxime Berube · Valve (United States)

Vanessa Bertone · Valve (United States)

Sarah Marie Cooper · Valve (United States)

Peter Dobrowolski · Valve (United States)

Helen Fong · Valve (United States)

Angela Genge · Valve (United States)

Matthew Hannouche · Valve (United States)

Denise Hartley · Valve (United States)

Michael Hogan · Valve (United States)

Wendy Johnston · Valve (United States)

Yousra Khalfallah · Valve (United States)

Lawrence Korngut · Valve (United States)

Gina Kroetsch · Valve (United States)

Justin Létourneau · Valve (United States)

Claire Magnussen · Valve (United States)

José Corberá Martínez · Valve (United States)

Rami Massie · Valve (United States)

Theodore Mobach · Valve (United States)

Jahan Mookshah · Valve (United States)

Timur Özelsel · Valve (United States)

Andrea Parks · Valve (United States)

Janet Petrillo · Valve (United States)

Gerald Pfeffer · Valve (United States)

Shirley Pham · Valve (United States)

Liane Phung · Valve (United States)

Rodney Shiungsun · Valve (United States)

Li Pi-Shan · Valve (United States)

Denizart Santos · Valve (United States)

Kristiana Salmon · Valve (United States)

Natalie Saunders · Valve (United States)

Dylan Sembinelli · Valve (United States)

Kelsey Tymkow · Valve (United States)

Berchman Wong · Valve (United States)

Lorne Zinman · Valve (United States)

Merete Karlsborg · Valve (United States)

Therese Pedersen Lomholt · Valve (United States)

Sigrid Nilsson · Valve (United States)

Lisette Salvesen · Valve (United States)

P. Stahl Skov · Valve (United States)

Kristen Svenstrup · Valve (United States)

Gaëlle Bruneteau · Valve (United States)

Frederic Clarencon · Valve (United States)

Raquel Guimarães Costa · Valve (United States)

Adèle Hesters · Valve (United States)

Hugo Kermorvant · Valve (United States)

Lucette Lacomblez · Valve (United States)

Nadine Le Forestier · Valve (United States)

Timothée Lenglet · Valve (United States)

Maryvonne Retail · Valve (United States)

M. del Mar Amador · Valve (United States)

François Salachas · Valve (United States)

Eimad Shotar · Valve (United States)

Nader Sourour · Valve (United States)

Johannes Dorst · Valve (United States)

Elke Verena Froehlich · Valve (United States)

Andrea Fromm · Valve (United States)

Katharina Kandler · Valve (United States)

Eva Langer · Valve (United States)

Sarah Leichtle · Valve (United States)

Ludolph Ac · Valve (United States)

Kristina Mayer · Valve (United States)

Sebastian Michels · Valve (United States)

Sabine Raubold · Valve (United States)

Joachim Schuster · Valve (United States)

Ulrike Weiland · Valve (United States)

Maximilian Wiesenfarth · Valve (United States)

Simon Witzel · Valve (United States)

Andrea Calvo · Valve (United States)

Antonio Canosa · Valve (United States)

Federico Casale · Valve (United States)

Adriano Chiò · Valve (United States)

Giuseppe Fuda · Valve (United States)

Maurizio Grassano · Valve (United States)

Giulia Marchese · Valve (United States)

Cristina Moglia · Valve (United States)

Francesca Palumbo · Valve (United States)

Paolina Salamone · Valve (United States)

Takahiro Ajiki · Valve (United States)

Aya Akasaka · Valve (United States)

Masahiro Ando · Valve (United States)

Hitoshi Arata · Valve (United States)

Kitamura Asuka · Valve (United States)

Kosuke Baba · Valve (United States)

Goichi Bekku · Valve (United States)

Tomoya Chiba · Valve (United States)

Yugaku Daté · Valve (United States)

Takeuchi Eriko · Valve (United States)

Akihiro Hashiguchi · Valve (United States)

Ritsuko Hatatori · Valve (United States)

Eri Hayano · Valve (United States)

Yuto Hayashi · Valve (United States)

Keiko Higashi · Valve (United States)

Eriko Higuchi · Valve (United States)

Yu Hiramatsu · Valve (United States)

Rui Horikawa · Valve (United States)

Kensuke Ikenaka · Valve (United States)

Hiroyuki Ishiura · Valve (United States)

Daisuke Ito · Valve (United States)

Sachiko Kawai · Valve (United States)

Junko Kikuchi · Valve (United States)

Haruko Kuzuyama · Valve (United States)

Xuehong Li · Valve (United States)

Matsumoto Cs · Valve (United States)

Eiji Matsuura · Valve (United States)

Kumiko Michizono · Valve (United States)

Jun Mitsui · Valve (United States)

Akihiko Mitsutake · Valve (United States)

Hiroyuki Mochizuki · Valve (United States)

Akemi Nagamatsu · Valve (United States)

S. Nagano · Valve (United States)

Tomonori Nakamura · Valve (United States)

Hiroya Naruse · Valve (United States)

Asuka Ogasawara · Valve (United States)

Kensuke Okada · Valve (United States)

Yuji Okamoto · Valve (United States)

Tatsusada Okuno · Valve (United States)

Satoshi Oyama · Valve (United States)

Tatsuhiko Ozono · Valve (United States)

Yusuke Sakiyama · Valve (United States)

Kaori Sakuishi · Valve (United States)

Morinobu Seki · Valve (United States)

Shota Shibata · Valve (United States)

Mikito Shimizu · Valve (United States)

Katsunori Takahata · Valve (United States)

Yoshizaki Takahito · Valve (United States)

Hiroshi Takashima · Valve (United States)

Hiroko Takeichi · Valve (United States)

Yuichi Tashiro · Valve (United States)

Tatsushi Toda · Valve (United States)

Yuki Tomizu · Valve (United States)

Wadayama Tomoya · Valve (United States)

Nishiike Ujiakira · Valve (United States)

Daiki Yashita · Valve (United States)

Ammar Al-Chalabi · Valve (United States)

James J. P. Alix · Valve (United States)

Priyadarshini Bangalore · Valve (United States)

Daniel Blackburn · Valve (United States)

Theresa Chiwera · Valve (United States)

Rosie Clegg · Valve (United States)

Alexis Collins · Valve (United States)

Johnathan Cooper-Knock · Valve (United States)

Anna T. Emery · Valve (United States)

John Franklin · Valve (United States)

Louisa Green · Valve (United States)

C Harvey · Valve (United States)

Esther Hobson · Valve (United States)

Mahjabim Islam · Valve (United States)

Thomas Michael Jenkins · Valve (United States)

Mbombe Kazoka · Valve (United States)

Gillian Kelly · Valve (United States)

Mercy Korley · Valve (United States)

Daniel Madarshahaian · Valve (United States)

Keith Mayl · Valve (United States)

Christopher John McDermott · Valve (United States)

Alex Radford · Valve (United States)

Christopher Shaw · Valve (United States)

Pamela J. Shaw · Valve (United States)

Joe Sidebottom · Valve (United States)

Lynne Smart · Valve (United States)

Jemeen Sreedharan · Valve (United States)

Ben Stone · Valve (United States)

Theocharis Tsironis · Valve (United States)

Lee Tuddenham · Valve (United States)

Nick Verber · Valve (United States)

Helen Wollff · Valve (United States)

Stacy Young · Valve (United States)

Panagiotis Zis · Valve (United States)

Ashley Adamo · Valve (United States)

Arubah Ahmed · Valve (United States)

Senda Ajroud-Driss · Valve (United States)

Gustave Alameda · Valve (United States)

Jinsy A. Andrews · Valve (United States)

Ximena Arcila-Londono · Valve (United States)

Suma Babu · Valve (United States)

Candy Baird · Valve (United States)

Tracy Bazan · Valve (United States)

Michael Benatar · Valve (United States)

James Berry · Valve (United States)

Jane Bordeau · Valve (United States)

Wendy Bradford · Valve (United States)

N R Brook · Valve (United States)

Lauren Brown · Valve (United States)

Robert C. Bucelli · Valve (United States)

Katherine Buckner · Valve (United States)

Michael W. Budler · Valve (United States)

Lindita Burba · Valve (United States)

Katherine Burke · Valve (United States)

Ashley Calhoun · Valve (United States)

Sarah Campbell · Valve (United States)

Judith Carey · Valve (United States)

Irys Caristo · Valve (United States)

Simon Carty · Valve (United States)

Emmanuel Chan · Valve (United States)

Vinay Chaudhry · Valve (United States)

Ricky Chen · Valve (United States)

Saephanh Chow · Valve (United States)

Lora L. Clawson · Valve (United States)

Mitchell Clemens · Valve (United States)

Suzann E. Cloninger · Valve (United States)

Krista Coleman-Wood · Valve (United States)

Thomas N. Cooper · Valve (United States)

Arlena Cummings · Valve (United States)

Jacquelyn Daniels · Valve (United States)

Pamela Desaro · Valve (United States)

Michelle DeWitt · Valve (United States)

Brixhilda Dedi · Valve (United States)

Debbie Dempsey · Valve (United States)

C. Dimla Denny · Valve (United States)

J Doherty · Valve (United States)

L. Doherty · Valve (United States)

M E Donahue · Valve (United States)

Michael Doyle · Valve (United States)

Jessie Duncan · Valve (United States)

Elman Lauren · Valve (United States)

Christine M. Eloge · Valve (United States)

Desirae R. Echiti · Valve (United States)

Dominic Ferrey · Valve (United States)

Christina Fournier · Valve (United States)

Yuriko Fukumura · Valve (United States)

Katherine Gallagher · Valve (United States)

Jessica Garaycoa · Valve (United States)

Mark Garrett · Valve (United States)

Richard L. Gibson · Valve (United States)

Gifford Rw · Valve (United States)

J. David Glass · Valve (United States)

D. Gogol · Valve (United States)

Shea R. Golden · Valve (United States)

Alexa Gonzalez · Valve (United States)

Ira Goodman · Valve (United States)

C. M. Goolsby · Valve (United States)

Kimberly Goslin · Valve (United States)

Michael Goulbourne · Valve (United States)

Volkan Granit · Valve (United States)

Anne-Laure Grignon · Valve (United States)

Adreeja GuhaRay · Valve (United States)

Debra Guide · Valve (United States)

Melek Betul Gundogdu · Valve (United States)

Gil Gutierrez · Valve (United States)

Debbie Hastings · Valve (United States)

Colleen Hayzen · Valve (United States)

Hilary Herzog · Valve (United States)

Raegan Holloway · Valve (United States)

Gabriel Jacobs · Valve (United States)

Bill Jacobsen · Valve (United States)

V. Nicole James · Valve (United States)

L. F. Jenkins · Valve (United States)

Jennifer Jockel-Balsarotti · Valve (United States)

Linda Carol Johnson · Valve (United States)

Sunil Jose · Valve (United States)

B F Joslin · Valve (United States)

Elizabeth Karanja · Valve (United States)

Jonathan Katz · Valve (United States)

Anthony Keener · Valve (United States)

Gale Kittle · Valve (United States)

Sara Klein · Valve (United States)

Collin J. Kreple · Valve (United States)

Rebecca Kuenzler · Valve (United States)

Jorge Kusnir · Valve (United States)

Kristen Labbe · Valve (United States)

Nicolet Lachica-Encinas · Valve (United States)

Shafeeq Ladha · Valve (United States)

Lesli Leimer · Valve (United States)

Michael Levy · Valve (United States)

Wendy Levy · Valve (United States)

Yingji Li · Valve (United States)

Marie-France N. Likanje · Valve (United States)

Rebecca Livigni · Valve (United States)

Eduardo Locatelli · Valve (United States)

Sarah Luppino · Valve (United States)

Amber Malcolm · Valve (United States)

Nicholas Maragakis · Valve (United States)

Horia Marin · Valve (United States)

Clyde Markowitz · Valve (United States)

Jesse Markway · Valve (United States)

Alexandra McCaffrey · Valve (United States)

Arita McCoy · Valve (United States)

Kelli Gross · Valve (United States)

Kush Mehta · Valve (United States)

Robert Meyer · Valve (United States)

Jennifer A. Milan · Valve (United States)

Timothy M. Miller · Valve (United States)

Robert G. Miller · Valve (United States)

Francisco R. Morales · Valve (United States)

Elizabeth Mosmiller · Valve (United States)

Donovan Mott · Valve (United States)

K S Moulton · Valve (United States)

Christine Murphy · Valve (United States)

Tirso Negron · Valve (United States)

Cassandra Nelson · Valve (United States)

Daniel S. Newman · Valve (United States)

Janne Kristoffer Nissinen · Valve (United States)

Andrew Norman · Valve (United States)

Takuya Ohkubo · Valve (United States)

Nicholas Olney · Valve (United States)

Natasha Ivelisse Roman Ortiz · Valve (United States)

Bjorn Oskarsson · Valve (United States)

Mitchell Pace · Valve (United States)

Kathleen A. Packard · Valve (United States)

Denny J. Padgett · Valve (United States)

Sabrina Paganoni · Valve (United States)

Maria Elena Paredes · Valve (United States)

Elizabeth Parker · Valve (United States)

Ann Partlow · Valve (United States)

Gary L. Pattee · Valve (United States)

Jany Paulett · Valve (United States)

Antoinette Pelot · Valve (United States)

Kyle M. Pfeifer · Valve (United States)

Olivia Pijanowski · Valve (United States)

E. Pioro · Valve (United States)

Meraida Polak · Valve (United States)

Ahalya Prakash · Valve (United States)

Rosemarie Previte · Valve (United States)

Bryan Pukenas · Valve (United States)

Colin C. Quinn · Valve (United States)

John Ravits · Valve (United States)

Ryan F. Razavi · Valve (United States)

Tyler Regan · Valve (United States)

Kristen M. Riley · Valve (United States)

Heather Roth · Valve (United States)

Danica Sanders · Valve (United States)

Jennifer Scalia · Valve (United States)

Emma Schmidt · Valve (United States)

Edward E. Schwen · Valve (United States)

Jaimin Shah · Valve (United States)

Stuti Shah · Valve (United States)

Jeremy Shefner · Valve (United States)

Danielle Sheldon · Valve (United States)

K W Simmons · Valve (United States)

Navneet K. Singh · Valve (United States)

Jessica Singleton · Valve (United States)

Richard Smiley · Valve (United States)

William B. Smith · Valve (United States)

Sean Smith · Valve (United States)

Elias Sotirchos · Valve (United States)

Eric Sorenson · Valve (United States)

Nathan Staff · Valve (United States)

Julie Steele · Valve (United States)

Kara Steijlen · Valve (United States)

Taylor Stirrat · Valve (United States)

George S. Stoica · Valve (United States)

Stephanie Strong · Valve (United States)

Robert Sufit · Valve (United States)

Jane Sultze · Valve (United States)

Amy Swartz · Valve (United States)

April L. Szymanski · Valve (United States)

Anna Tay · Valve (United States)

Nimish J. Thakore · Valve (United States)

Diana Thiessen · Valve (United States)

Sukrutha Thotala · Valve (United States)

Randall G. Trudell · Valve (United States)

Nicole Turcotte · Valve (United States)

Michelle Turner · Valve (United States)

Alpa Uchil · Valve (United States)

Vihar Upadhyay · Valve (United States)

U. Usman · Valve (United States)

Anne Vallis · Valve (United States)

Danielle Vaporean-Bussey · Valve (United States)

Valentine Vladimirova · Valve (United States)

Harli Weber · Valve (United States)

Jennifer Winbigler · Valve (United States)

Heather Wojanowski · Valve (United States)

Charlie Wulf · Valve (United States)

Julia Yasek · Valve (United States)

Stephanie Yoo · Valve (United States)

Hannah Zivalic · Valve (United States)

Alexandra Cole · Valve (United States)

Greta File · Valve (United States)

J. A. Foate · Valve (United States)

Deborah L. Mason · Valve (United States)

S. Newton · Valve (United States)

Pu Pu · Valve (United States)

Cory Dean Sellwood · Valve (United States)

James Swan · Valve (United States)

Anja Werno · Valve (United States)

Cathy S. Zhong · Valve (United States)