Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My Custom Filters

Edit custom filters

Results by year

Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2012 1
2020 1
2021 1
2022 5
2023 12
2024 26
2025 30
2026 21

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

86 results

Results by year

Citations

10 articles found by citation matching
Show all

Search results

Filters applied: . Clear all
Page 1
Tofersen for SOD1 amyotrophic lateral sclerosis: a systematic review and meta-analysis.
Hamad AA, Alkhawaldeh IM, Nashwan AJ, Meshref M, Imam Y. Hamad AA, et al. Neurol Sci. 2025 May;46(5):1977-1985. doi: 10.1007/s10072-025-07994-2. Epub 2025 Jan 17. Neurol Sci. 2025. PMID: 39820998 Free PMC article.
OBJECTIVE: Tofersen, an antisense oligonucleotide, has recently received FDA and EMA approval for treating amyotrophic lateral sclerosis (ALS) in adults with SOD1 gene mutations. ...CONCLUSION: Current evidence suggests that tofersen effe …
OBJECTIVE: Tofersen, an antisense oligonucleotide, has recently received FDA and EMA approval for treating amyotrophic late …
Tofersen: A Review in Amyotrophic Lateral Sclerosis Associated with SOD1 Mutations.
McGuigan A, Blair HA. McGuigan A, et al. CNS Drugs. 2025 Sep;39(9):903-912. doi: 10.1007/s40263-025-01204-5. Epub 2025 Jul 10. CNS Drugs. 2025. PMID: 40640528 Review.
Tofersen (QALSODY( )) is the first drug approved for the treatment of amyotrophic lateral sclerosis (ALS) associated with superoxide dismutase 1 (SOD1) mutations. ...Amyotrophic lateral sclerosis (ALS) is a fatal neurodegene …
Tofersen (QALSODY( )) is the first drug approved for the treatment of amyotrophic lateral sclerosis (ALS) associ …
Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis.
Miller TM, Cudkowicz ME, Shaw PJ, Genge A, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Salachas F, Bruneteau G, Al-Chalabi A, Amorin M, Nestorov I, Graham D, Lin L, Sun P, McNeill M, Malek S, Inra J, Garafalo S, Fradette S; VALOR and OLE Working Group. Miller TM, et al. JAMA Neurol. 2026 Feb 1;83(2):115-125. doi: 10.1001/jamaneurol.2025.4946. JAMA Neurol. 2026. PMID: 41661214 Free PMC article. Clinical Trial.
IMPORTANCE: Approximately 2% of amyotrophic lateral sclerosis (ALS) cases are attributable to a pathogenic variant in the superoxide dismutase 1 (SOD1) gene. ...Over 148 weeks, earlier initiation of tofersen (compared to later initiation) was as …
IMPORTANCE: Approximately 2% of amyotrophic lateral sclerosis (ALS) cases are attributable to a pathogenic variant in t …
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S; VALOR and OLE Working Group. Miller TM, et al. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705. N Engl J Med. 2022. PMID: 36129998 Free article. Clinical Trial.
BACKGROUND: The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with amyotrophic lateral sclerosis (ALS) associated with mutations in …
BACKGROUND: The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD …
Tofersen for SOD1 ALS.
Everett WH, Bucelli RC. Everett WH, et al. Neurodegener Dis Manag. 2024;14(5):149-160. doi: 10.1080/17582024.2024.2402216. Epub 2024 Sep 27. Neurodegener Dis Manag. 2024. PMID: 39330700 Free PMC article. Review.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative condition affecting the motor system. ...Tofersen is a recently approved medication that acts by reducing a toxic protein found in a particular form of inherited amyotrophic lateral …
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative condition affecting the motor system. ...Tofersen is …
Amyotrophic Lateral Sclerosis and Other Motor Neuron Diseases.
Izenberg A. Izenberg A. Continuum (Minneap Minn). 2023 Oct 1;29(5):1538-1563. doi: 10.1212/CON.0000000000001345. Continuum (Minneap Minn). 2023. PMID: 37851042 Review.
OBJECTIVE: This article reviews the clinical spectrum of amyotrophic lateral sclerosis (ALS), its variant presentations, and the approach to diagnosis and management. ...An oral formulation of edaravone and an antisense oligonucleotide to a SOD1 gene v …
OBJECTIVE: This article reviews the clinical spectrum of amyotrophic lateral sclerosis (ALS), its variant presentations …
Antisense oligonucleotide therapy in amyotrophic lateral sclerosis.
Erdi-Krausz G, Shaw PJ. Erdi-Krausz G, et al. Curr Opin Neurol. 2025 Oct 1;38(5):574-580. doi: 10.1097/WCO.0000000000001413. Epub 2025 Aug 20. Curr Opin Neurol. 2025. PMID: 40832750 Free article. Review.
PURPOSE OF REVIEW: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder with few treatment options available. The approval of tofersen, an antisense oligonucleotide, for SOD1 -ALS by the FDA and EMA may herald a new era of t …
PURPOSE OF REVIEW: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder with few treatment options …
ASO drug Qalsody (tofersen) targets amyotrophic lateral sclerosis.
Jin J, Zhong XB. Jin J, et al. Trends Pharmacol Sci. 2023 Dec;44(12):1043-1044. doi: 10.1016/j.tips.2023.08.008. Epub 2023 Sep 12. Trends Pharmacol Sci. 2023. PMID: 37709589 Free PMC article.
[Image: see text] MECHANISM OF ACTION: (A) Approximately 2% of cases of amyotrophic lateral sclerosis (ALS) in adults is caused by a genetic mutation in the superoxide dismutase 1 (SOD1) gene. ...(B) Tofersen is indicated for the treatment of AL …
[Image: see text] MECHANISM OF ACTION: (A) Approximately 2% of cases of amyotrophic lateral sclerosis (ALS) in adults i …
Tofersen in SOD1-associated amyotrophic lateral sclerosis: From molecular mechanisms to regulatory milestones.
Braza AJ, Viñas-Bastart M, Sureda-Rosich M, García-Parra B, Guiu-Segura JM, Modamio P. Braza AJ, et al. Eur J Pharm Sci. 2026 May 21;223:107563. doi: 10.1016/j.ejps.2026.107563. Online ahead of print. Eur J Pharm Sci. 2026. PMID: 42173382 Free article. Review.
BACKGROUND: Amyotrophic Lateral Sclerosis (ALS) is a progressive and ultimately fatal neurodegenerative disorder characterized by degeneration of upper and lower motor neurons. ...Tofersen is an antisense oligonucleotide therapy designed to reduce the …
BACKGROUND: Amyotrophic Lateral Sclerosis (ALS) is a progressive and ultimately fatal neurodegenerative disorder charac …
86 results