Long-term treatment of SOD1 ALS with tofersen: a multicentre experience in 17 patients-Reference-Cited by-同舟云学术

Long-term treatment of SOD1 ALS with tofersen: a multicentre experience in 17 patients

Published:2024-06-03 Issue:8 Volume:271 Page:5177-5186
ISSN:0340-5354
Container-title:Journal of Neurology
language:en
Short-container-title:J Neurol

Author:

Sabatelli Mario^ORCID,Cerri Federica,Zuccarino Riccardo,Patanella Agata Katia,Bernardo Daniela,Bisogni Giulia,Tanel Raffaella,Sansone Valeria,Filosto Massimiliano,Lattante Serena,Martello Francesco,Doronzio Paolo Niccolò,Stano Salvatore,Zanfini Bruno Antonio,Coccia Michela,Costantini Emanuele Maria,Lizio Andrea,Lucioli Gabriele,Padovani Alessandro,Merlini Gian Paolo,Conte Amelia

Publisher

Springer Science and Business Media LLC

Link

https://link.springer.com/content/pdf/10.1007/s00415-024-12437-7.pdf

Reference9 articles.

1. Müller K, Brenner D, Weydt P et al (2018) Comprehensive analysis of the mutation spectrum in 301 German ALS families. J Neurol Neurosurg Psychiatry 89:817–827. https://doi.org/10.1136/jnnp-2017-317611

2. Saccon RA, Bunton-Stasyshyn RK, Fisher EM, Fratta P (2013) Is SOD1 loss of function involved in amyotrophic lateral sclerosis? Brain 136:2342–2358. https://doi.org/10.1093/brain/awt097

3. McCampbell A, Cole T, Wegener AJ et al (2018) Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models. J Clin Invest 128:3558–3567. https://doi.org/10.1172/JCI99081

4. Miller T, Cudkowicz M, Shaw PJ et al (2020) Phase 1–2 trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med 383:109–119. https://doi.org/10.1056/NEJMoa2003715

5. Miller TM, Cudkowicz ME, Genge A et al (2022) Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med 387:1099–1110. https://doi.org/10.1056/NEJMoa2204705

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1. SOD1 gene screening in ALS – frequency of mutations, patients’ attitudes to genetic information and transition to tofersen treatment in a multi-center program;Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration;2024-09-13

2. Changes in Cerebrospinal Fluid Concentrations of Selenium Species Induced by Tofersen Administration in Subjects with Amyotrophic Lateral Sclerosis Carrying SOD1 Gene Mutations;Biological Trace Element Research;2024-07-17