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References from Towards the integration of open-label extension and real-world evidence in ALS therapeutic evaluation?. Local targets link to admitted publications; unresolved targets remain external evidence.
Trial of antisense oligonucleotide tofersen for SOD1 ALS
10.1056/nejmoa2204705 · 2022 · External reference
Tofersen for SOD1 amyotrophic lateral sclerosis: a systematic review and meta-analysis
10.1007/s10072-025-07994-2 · 2025 · External reference
Tofersen treatment in SOD1-ALS: real-world evidence from a retrospective multicenter study in France (FORSLA Study)
10.1016/j.mayocpiqo.2026.100739 · 2026 · External reference
Tofersen and other antisense oligonucleotides in ALS
10.1177/17562864251313915 · 2025 · External reference
Long-term tofersen in SOD1 amyotrophic lateral sclerosis
10.1001/jamaneurol.2025.4946 · 2026 · External reference
Tofersen decreases neurofilament levels supporting the pathogenesis of the SOD1 p. D91A variant in amyotrophic lateral sclerosis patients
10.1038/s43856-024-00573-0 · 2024 · External reference
Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS – A multicenter observational study over 18 months
10.1002/mus.28182 · 2024 · External reference
Prevalence of SOD1 and C9orf72 variants among French ALS Population: The GENIALS Study
10.1111/ene.70302 · 2025 · External reference
