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| 001 | 276161 | ||
| 005 | 20250209000726.0 | ||
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| 037 | _ | _ | |a DZNE-2025-00233 |
| 041 | _ | _ | |a English |
| 082 | _ | _ | |a 610 |
| 100 | 1 | _ | |a Ludolph, Albert |0 P:(DE-2719)2812633 |b 0 |e First author |u dzne |
| 245 | _ | _ | |a Tofersen and other antisense oligonucleotides in ALS. |
| 260 | _ | _ | |a London [u.a.] |c 2025 |b Sage |
| 336 | 7 | _ | |a article |2 DRIVER |
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| 520 | _ | _ | |a The advent of antisense oligonucleotide (ASO) therapies in neurodegenerative disorders is associated with enormous hope. Nusinersen treatment was a breakthrough intervention in the recessive disease spinal muscular atrophy, and superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) seems to be the paradigm disease in dominant degenerative diseases. The results of treatment with the ASO tofersen in SOD1-ALS show that the drug has a convincing beneficial effect on ALS caused by SOD1 mutations, that preclinical studies in rodents predicted the therapeutic effect in the human disease, and that clinical efficacy is associated with a specific sequence of effects of the drug on mechanistic and degenerative biomarkers and, subsequently, functional outcomes such as weight stabilization and ALSFRS-R. Therefore, the enthusiasm seems to be justified; but this should be followed by an attempt to obtain further insights with the goal to improve this therapy. In particular, the following issues are only partially resolved: Which mechanisms are responsible for the clinical effect following the downregulation of SOD1 protein by ASOs? Is long-term downregulation of SOD1 function associated with side effects? Is there an autoimmune response caused by this and other ASO? Is prevention of SOD1-associated ALS possible? |
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| 650 | _ | 7 | |a amyotrophic lateral sclerosis |2 Other |
| 650 | _ | 7 | |a antisense oligonucleotides |2 Other |
| 650 | _ | 7 | |a superoxide dismutase |2 Other |
| 650 | _ | 7 | |a tofersen |2 Other |
| 700 | 1 | _ | |a Wiesenfarth, Maximilian |b 1 |
| 773 | _ | _ | |a 10.1177/17562864251313915 |g Vol. 18, p. 17562864251313915 |0 PERI:(DE-600)2442245-9 |p 17562864251313915 |t Therapeutic advances in neurological disorders |v 18 |y 2025 |x 1756-2856 |
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