The first part of Biogen’s early access program for tofersen is now open to patients with the most rapidly progressing cases of familial amyotrophic lateral sclerosis (ALS), the company announced. These patients, with familial ALS caused by mutations in the SOD1 gene, will be the first to…
Exservan, the first oral film formulation of riluzole for treating amyotrophic lateral sclerosis (ALS), has been launched in the United States, its developer, Mitsubishi Tanabe Pharma America (MTPA), has announced. Rapidly dissolved when placed on top of the tongue, Exservan — specifically developed to help meet the needs…
Amylyx Pharmaceuticals is seeking approval, in Canada, of its lead candidate AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). This regulatory submission “represents a significant milestone in our efforts to develop a new treatment option for people living with ALS who have no time to wait,” Joshua Cohen, co-founder, co-CEO,…
At face value, the purpose of the National Amyotrophic Lateral Sclerosis (ALS) Registry seems quite straightforward: collecting data on the U.S. population with ALS. But there’s a lot more to it. The registry, funded by the…
Cytokinetics has reaffirmed its partnership with The ALS Association in the fight against amyotrophic lateral sclerosis (ALS) by continuing to support events and initiatives throughout 2021. The muscle biology biopharmaceutical company also is working to advance the clinical development of its experimental therapy reldesemtiv, designed to improve…
SOL-257, an experimental gene therapy developed by SOLA Biosciences, delayed disease progression and prolonged the lifespan of a mouse model of amyotrophic lateral sclerosis (ALS). The gene therapy is designed to reduce the abnormal TDP-43 protein that forms toxic clumps in the cells of ALS patients. “Targeting only…
A Phase 3 clinical trial evaluating Orphazyme’s investigational oral therapy arimoclomol for amyotrophic lateral sclerosis (ALS) has failed to meet its primary and key secondary goals. While no safety issues were reported in the trial (NCT03491462), arimoclomol did not significantly extend patients’ lives nor…
Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. In a Phase 1 trial for amyotrophic lateral sclerosis (ALS), the investigational therapy AP-101 — designed to ease…
Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. WVE-004, an investigational treatment for people with amyotrophic lateral sclerosis…
The HEALEY ALS platform trial, which is evaluating the safety and efficacy of several potential treatments for amyotrophic lateral sclerosis (ALS), has passed the 50% participant enrollment target for its first three regimens. The investigational treatments are UCB’s zilucoplan, Biohaven Pharmaceuticals’ verdiperstat, and Clene Nanomedicine’s CNM-Au8. A fourth candidate…
