Patricia Inácio, PhD, science writer —

Aquinnah Pharmaceuticals has partnered with Roche to further develop its brain-penetrating, oral small molecules for the treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. These small molecules are designed to eliminate the persistent stress granules that contribute to the buildup of toxic protein clumps in…

The investigational antibody therapy for amyotrophic lateral sclerosis (ALS), AT-1501, will now be called tegoprubart, its maker, Eledon Pharmaceuticals, has announced. The change comes after the United States Adopted Names (USAN) Council selected tegoprubart as the unique generic, or nonproprietary, name for the therapy candidate. The company…

A mouse model of amyotrophic lateral sclerosis (ALS) carrying a mutation in the SOD1 gene showed alterations in the gut microbiome, followed by motor impairments and defects in enteric nervous system — the gut’s own autonomous nervous system — compared with healthy mice, a study found. These microbiome changes occurred…

Eikonoklastes Therapeutics has added a candidate gene therapy for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases to its pipeline. The announcement follows the completion of a licensing agreement with the University of California San Diego. Brian Head, PhD, a professor with the university’s department of anesthesiology, and…

Amyotrophic lateral sclerosis (ALS) patients who completed the expanded access protocol (EAP) for NurOwn may soon be eligible for three additional doses of the cell-based therapy, BrainStorm Cell Therapeutics reported. EAPs, also known as compassionate use programs, are intended to make investigational therapies available outside of a clinical trial…

Amydis announced plans to launch a first clinical trial next year into the ability of its small molecule tracer to detect toxic TDP-43 clumps — a hallmark of amyotrophic lateral sclerosis (ALS) — in the eye’s retina. Its announcement follows a preliminary meeting with U.S. Food and Drug Administration…

A Phase 2 trial of ZZ Biotech’s investigational therapy 3K3A-APC in people with amyotrophic lateral sclerosis (ALS) has started dosing patients at its single site in Australia, the company announced in a press release. The open-label trial (NCT05039268), underway at the Macquarie University in Sydney, aims to evaluate…

CNM-Au8, an investigational disease-modifying therapy for people with early amyotrophic lateral sclerosis (ALS), showed consistent clinical benefits in patients with both limb and bulbar onset disease, according to new analyses of RESCUE-ALS Phase 2 clinical trial data. In particular, the treatment significantly extended the time to disease progression —…

Brainstorm Cell Therapeutics‘ investigational cell-based therapy, NurOwn, might be able to slow disease progression and be of “meaningful” benefit to  people with less severe amyotrophic lateral sclerosis (ALS), according to new analyses of Phase 3 trial data. The company announced last year that its Phase 3 trial…

Prilenia Therapeutics has raised $43 million to support potential regulatory submissions and marketing of its lead candidate, pridopidine, for the treatment of amyotrophic lateral sclerosis (ALS). The funding, obtained through a Series B financing round, also will be used to support the potential marketing of the candidate therapy for…