QALSODY™ - ALZFORUM
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 mechanism remains unknown, mutant SOD1 is believed to exert a toxic action on motor neurons, and reducing its levels may be beneficial.
Windfall Week for Trial Data: Papers on Crenezumab, …
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 72 people dosed for 28 weeks. Nonetheless, the company, with its partner Ionis Pharmaceuticals, forged ahead and applied to the FDA for accelerated approval.
Neurofilament Light Clears First Hurdle as Bona Fide FTD Biomarker
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 timescale of a clinical trial, the Bluefield Neurofilament Surveillance Program is measuring plasma NfL every three months for three years among more 335 familial FTD ...
FDA Grants Accelerated Approval for Tofersen - ALZFORUM
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 a drop in a biomarker, in this case levels of neurofilament light chain in the plasma. Tofersen did not slow clinical decline in a Phase 3 study (Oct 2021 news). Over the ...
Antisense Oligonucleotides: Can They Take on ALS, SMA, Prions?
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 randomized to receive either 20, 40, 60, or 100 mg tofersen or placebo. There were 10, nine, nine, 10, and 12 people on each treatment, respectively.
Tofersen: First Approval. | ALZFORUM
Blair HA.Tofersen: First Approval. Drugs. 2023 Jul;83(11):1039-1043. PubMed.. Recommends. Please login to recommend the paper.
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
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 …
Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study. Neurotherapeutics. 2022 Jul;19(4):1248-1258. Epub 2022 May 18 PubMed. Recommends. Please login to recommend the paper. Comments. No Available Comments. Make a Comment. To make a comment you must ...
Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 …
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. No Available Comments. Make a Comment. To make a comment you must login or register.
Clinical and patient-reported outcomes and neurofilament …
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 Jun 20; PubMed. Recommends. Please login to recommend the paper. Comments. No Available Comments. Make a Comment. To make a comment you must login or register.