001     280914
005     20250921001947.0
024 7 _ |a 10.1002/mus.28453
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037 _ _ |a DZNE-2025-00997
041 _ _ |a English
082 _ _ |a 610
100 1 _ |a Bernsen, Sarah
|0 P:(DE-2719)9000485
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245 _ _ |a Serum Cardiac Troponin T Levels as a Therapy Response Marker in Tofersen-Treated ALS.
260 _ _ |a New York, NY [u.a.]
|c 2025
|b Wiley
336 7 _ |a article
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520 _ _ |a Cardiac troponin T (cTnT) levels are elevated in the majority of persons with amyotrophic lateral sclerosis (ALS) and increase over time. Neurofilament light chain (NfL) is an established therapy response biomarker in ALS as superoxide dismutase1 (SOD1)-ALS patients treated with the antisense oligonucleotide tofersen show a decrease in NfL. In this study, we assess cTnT levels in SOD1-ALS at baseline and during tofersen treatment.cTnT was analyzed at baseline and during tofersen treatment in 23 SOD1-ALS patients at two specialized ALS centers in Germany and compared to a control cohort of 74 ALS patients without SOD1 variants.cTnT levels increased in the control ALS cohort over time (p < 0.0001) but not in the tofersen group (p = 0.36). Creatine kinase (CK) and CK-MB levels did not show significant changes over time. The median monthly increase of cTnT was 0.045 points (IQR 0.02-0.08) in the control ALS cohort and 0.01 points (IQR -0.01-0.03) in the tofersen group (p = 0.0013). A significantly lower fold change in cTnT levels was observed in the tofersen-treated cohort (median 1.2; IQR 0.77-1.59) relative to the control group (median 1.89; IQR 1.35-2.75) (p = 0.0003). Nine (39%) patients treated with tofersen experienced a reduction in cTnT levels.In this study, we describe a response signal of cTnT to tofersen treatment, which supports the value of cTnT as an independent biomarker in ALS. These results contribute to the notion that cTnT may provide additional value as a progression and treatment response biomarker in ALS complementary to NfL and warrant further investigation.
536 _ _ |a 353 - Clinical and Health Care Research (POF4-353)
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650 _ 7 |a SOD1
|2 Other
650 _ 7 |a amyotrophic lateral sclerosis
|2 Other
650 _ 7 |a biomarker
|2 Other
650 _ 7 |a tofersen
|2 Other
650 _ 7 |a troponin
|2 Other
650 _ 7 |a Troponin T
|2 NLM Chemicals
650 _ 7 |a Biomarkers
|2 NLM Chemicals
650 _ 7 |a Oligonucleotides
|2 NLM Chemicals
650 _ 7 |a Neurofilament Proteins
|2 NLM Chemicals
650 _ 7 |a Superoxide Dismutase-1
|0 EC 1.15.1.1
|2 NLM Chemicals
650 _ 7 |a Creatine Kinase
|0 EC 2.7.3.2
|2 NLM Chemicals
650 _ 7 |a neurofilament protein L
|2 NLM Chemicals
650 _ 2 |a Humans
|2 MeSH
650 _ 2 |a Amyotrophic Lateral Sclerosis: blood
|2 MeSH
650 _ 2 |a Amyotrophic Lateral Sclerosis: drug therapy
|2 MeSH
650 _ 2 |a Amyotrophic Lateral Sclerosis: genetics
|2 MeSH
650 _ 2 |a Troponin T: blood
|2 MeSH
650 _ 2 |a Male
|2 MeSH
650 _ 2 |a Female
|2 MeSH
650 _ 2 |a Middle Aged
|2 MeSH
650 _ 2 |a Aged
|2 MeSH
650 _ 2 |a Biomarkers: blood
|2 MeSH
650 _ 2 |a Oligonucleotides: therapeutic use
|2 MeSH
650 _ 2 |a Treatment Outcome
|2 MeSH
650 _ 2 |a Neurofilament Proteins
|2 MeSH
650 _ 2 |a Superoxide Dismutase-1: genetics
|2 MeSH
650 _ 2 |a Cohort Studies
|2 MeSH
650 _ 2 |a Adult
|2 MeSH
650 _ 2 |a Creatine Kinase: blood
|2 MeSH
700 1 _ |a Fabian, Rachel
|b 1
700 1 _ |a Koc, Yasemin
|b 2
700 1 _ |a Schumann, Peggy
|b 3
700 1 _ |a Körtvélyessy, Peter
|0 P:(DE-2719)2812030
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700 1 _ |a Castro-Gomez, Mario Sergio
|0 P:(DE-2719)9002230
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700 1 _ |a Meyer, Thomas
|0 0000-0002-2736-7350
|b 6
700 1 _ |a Weydt, Patrick
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773 _ _ |a 10.1002/mus.28453
|g Vol. 72, no. 3, p. 509 - 514
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|t Muscle & nerve
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