- 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
- Antisense Oligonucleotides: Can They Take on ALS, SMA, Prions?
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
- FDA Grants Accelerated Approval for Tofersen - ALZFORUM
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
- Neurofilament Light Clears First Hurdle as Bona Fide FTD Biomarker
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
- Long-term treatment of SOD1 ALS with tofersen: a multicentre experience . . .
Long-term treatment of SOD1 ALS with tofersen: a multicentre experience in 17 patients J Neurol 2024 Aug;271(8):5177-5186 Epub 2024 Jun 3 PubMed Recommends Please login to recommend the paper Comments No Available Comments Make a Comment To make a comment you must login or register
- Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. | ALZFORUM
Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS N Engl J Med 2022 Sep 22;387(12):1099-1110
- Windfall Week for Trial Data: Papers on Crenezumab, Neflamapimod, Tofersen
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
- Tofersen: First Approval. | ALZFORUM
Blair HA Tofersen: First Approval Drugs 2023 Jul;83(11):1039-1043 PubMed Recommends Please login to recommend the paper
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