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@ARTICLE{Ludolph:276161,
      author       = {Ludolph, Albert and Wiesenfarth, Maximilian},
      title        = {{T}ofersen and other antisense oligonucleotides in {ALS}.},
      journal      = {Therapeutic advances in neurological disorders},
      volume       = {18},
      issn         = {1756-2856},
      address      = {London [u.a.]},
      publisher    = {Sage},
      reportid     = {DZNE-2025-00233},
      pages        = {17562864251313915},
      year         = {2025},
      abstract     = {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?},
      subtyp        = {Review Article},
      keywords     = {amyotrophic lateral sclerosis (Other) / antisense
                      oligonucleotides (Other) / superoxide dismutase (Other) /
                      tofersen (Other)},
      cin          = {Clinical Study Center (Ulm)},
      ddc          = {610},
      cid          = {I:(DE-2719)5000077},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:39845577},
      pmc          = {pmc:PMC11752197},
      doi          = {10.1177/17562864251313915},
      url          = {https://pub.dzne.de/record/276161},
}