{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T10:13:45Z","timestamp":1788257625900,"version":"build-2803163510"},"reference-count":40,"publisher":"SAGE Publications","issue":"8","license":[{"start":{"date-parts":[[2026,2,4]],"date-time":"2026-02-04T00:00:00Z","timestamp":1770163200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Ann Pharmacother"],"published-print":{"date-parts":[[2026,8]]},"abstract":"<jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>This review summarizes current evidence on the efficacy and safety of tofersen (Qalsody) in treating amyotrophic lateral sclerosis (ALS).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Data Sources:<\/jats:title>\n                    <jats:p>\n                      PubMed, MEDLINE, Google Scholar, and ClinicalTrials.gov were searched using the keywords:\n                      <jats:italic toggle=\"yes\">Qalsody<\/jats:italic>\n                      ,\n                      <jats:italic toggle=\"yes\">BIIB067<\/jats:italic>\n                      ,\n                      <jats:italic toggle=\"yes\">antisense oligonucleotides<\/jats:italic>\n                      ,\n                      <jats:italic toggle=\"yes\">SOD1<\/jats:italic>\n                      , and\n                      <jats:italic toggle=\"yes\">amyotrophic lateral sclerosis<\/jats:italic>\n                      . Articles published from inception to November 2025 were included.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Study Selection and Data Extraction:<\/jats:title>\n                    <jats:p>English-language studies assessing the pharmacokinetics, pharmacology, efficacy, and safety of tofersen were included. Prescribing information and real-world evidence were also reviewed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Data Synthesis:<\/jats:title>\n                    <jats:p>\n                      Tofersen is an intrathecally administered antisense oligonucleotide targeting superoxide dismutase 1 (SOD1) mRNA. Early trials demonstrate dose-dependent reductions in cerebrospinal fluid (CSF) SOD1 protein levels of \u221233% and slower ALS Functional Rating Scale (ALSFRS-R) decline compared to placebo (\u22121.19 vs \u22125.63 points). In Phase 3 trials, tofersen reduced CSF SOD1 by 29% and plasma neurofilament light chain (NfL) by 60%, while biomarkers increased in the placebo group. There was no significant difference in ALSFRS-R decline between tofersen and placebo (\u22126.98 vs \u22128.14;\n                      <jats:italic toggle=\"yes\">P<\/jats:italic>\n                      = 0.97). Real-world data show favorable patient-related outcomes and improvement in ALSFRS-R. Adverse effects are primarily lumbar puncture related with serious neurologic events documented in 7% of tofersen recipients.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Relevance to Patient Care and Clinical Practice in Comparison to Existing Drugs:<\/jats:title>\n                    <jats:p>As the first Food and Drug Administration (FDA)-approved gene-directed therapy for SOD1 ALS, tofersen directly targets the underlying genetic cause. Barriers include the need for genetic confirmation and intrathecal administration.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Tofersen provides a promising targeted treatment option for pathogenic SOD1 ALS. Ongoing studies will clarify its long-term clinical impact.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/10600280251408862","type":"journal-article","created":{"date-parts":[[2026,2,5]],"date-time":"2026-02-05T06:37:31Z","timestamp":1770273451000},"page":"795-804","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":1,"title":["Tofersen: A Novel Option for the Treatment of Amyotrophic Lateral Sclerosis"],"prefix":"10.1177","volume":"60","author":[{"given":"Michael","family":"Karros","sequence":"first","affiliation":[{"name":"Fairleigh Dickinson University College of Pharmacy and Health Sciences, Florham Park, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marissa","family":"DiFulco","sequence":"additional","affiliation":[{"name":"Fairleigh Dickinson University College of Pharmacy and Health Sciences, Florham Park, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1391-2178","authenticated-orcid":false,"given":"Anna","family":"Nogid","sequence":"additional","affiliation":[{"name":"Fairleigh Dickinson University College of Pharmacy and Health Sciences, Florham Park, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,2,4]]},"reference":[{"key":"e_1_3_2_2_2","doi-asserted-by":"publisher","DOI":"10.3390\/ijms20112818"},{"key":"e_1_3_2_3_2","doi-asserted-by":"publisher","DOI":"10.1097\/WCO.0000000000000730"},{"key":"e_1_3_2_4_2","doi-asserted-by":"publisher","DOI":"10.3109\/21678421.2015.1074707"},{"key":"e_1_3_2_5_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000006317"},{"key":"e_1_3_2_6_2","doi-asserted-by":"publisher","DOI":"10.3390\/genes15101342"},{"key":"e_1_3_2_7_2","doi-asserted-by":"publisher","DOI":"10.1038\/nn.3584"},{"key":"e_1_3_2_8_2","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.52264"},{"key":"e_1_3_2_9_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2204705"},{"key":"e_1_3_2_10_2","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16264"},{"key":"e_1_3_2_11_2","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-022-01769-1"},{"key":"e_1_3_2_12_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199403033300901"},{"issue":"1","key":"e_1_3_2_13_2","first-page":"25","article-title":"Edaravone (radicava)","volume":"43","author":"Cruz MP.","year":"2018","unstructured":"Cruz MP. 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