Tofersen is being developed for ALS caused by SOD1 mutations, which account for about 20 percent of all familial ALS and 2 percent of all ALS cases. Although the exact pathological …

Sep 23, 2022 · Tofersen hit its target, successfully reducing SOD1 protein levels in CSF, but did not significantly stanch decline on the ALS Functional Rating Scale as compared to placebo in …

May 28, 2024 · That said, scientists already have their sights set on qualifying NfL as an endpoint in Phase 3 clinical trials, as well, a lá tofersen. To understand how NfL fluctuates on the …

Apr 26, 2023 · As with the approval of Biogen’s aducanumab for Alzheimer’s disease almost two years ago, the accelerated approval for tofersen was based not on slowing of disease, but on …

May 22, 2019 · The trial tested tofersen in ALS patients with confirmed SOD1 mutations. In Philadelphia, Miller presented its multiple-ascending-dose portion. Fifty participants were …

Blair HA.Tofersen: First Approval. Drugs. 2023 Jul;83(11):1039-1043. PubMed.. Recommends. Please login to recommend the paper.

Apr 26, 2023 · Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022 Sep 22;387(12):1099-1110.

Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study. Neurotherapeutics. 2022 …

Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2020 Jul 9;383(2):109-119. PubMed. Recommends. Please login to recommend the paper. Comments. …

Clinical and patient-reported outcomes and neurofilament response during tofersen treatment in SOD1-related ALS-A multicenter observational study over 18 months. Muscle Nerve. 2024 …