Lindsey Shapiro, PhD,  science writer—

Brainstorm Cell Therapeutics is moving forward in its efforts to get needed regulatory clearances to launch a Phase 3b clinical trial in the U.S. to test its stem cell therapy NurOwn (debamestrocel) in people with amyotrophic lateral sclerosis (ALS) with less advanced disease. The company has submitted to…

Trace Neuroscience plans to test genomic medicine that targets the UNC13A protein in people with amyotrophic lateral sclerosis (ALS) in a Phase 1/2 clinical trial that will be supported by artificial intelligence (AI)-powered tools from Unlearn. Unlearn’s Digital Twin Generator for ALS (ALS DTG) will be used…

NUZ-001, which was previously known as monepantel, slowed lung function declines by nearly 50% in people with amyotrophic lateral sclerosis (ALS), according to new analyses from a Phase 1 trial. This slowing of respiratory declines was strongly correlated with slower declines in overall function that were observed in…

Certain subsets of natural killer (NK) cells, a part of the immune system, were elevated in the blood of people with amyotrophic lateral sclerosis (ALS), where they showed altered gene activity and signaling patterns, a study found. One subset was associated with altered immune signaling, while another was linked…

Signaling at the low-density lipoprotein receptor, known as LDLR, may be an important driver of neurodegeneration in sporadic amyotrophic lateral sclerosis (ALS), according to new preclinical research by U.S. scientists. In earlier work, the scientists discovered that mice injected with cerebrospinal fluid, or CSF — the fluid that…

A single dose of Insmed’s investigational gene therapy INS1202 preserved motor function and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations. The therapy was also found to protect against nerve cell loss and had other beneficial effects on disease-related biomarkers.

Treatment with CNM-Au8 in the HEALEY ALS platform trial prolonged survival for people with amyotrophic lateral sclerosis (ALS) relative to those who received the investigational treatment zilucoplan or a placebo in a separate arm of the study, according to new analyses. The benefits were most pronounced in the…

The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the…

Health Canada has conditionally approved Qalsody (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. Qalsody, designed to lower levels of the toxic protein that drives the disease for these patients, is the first treatment to directly target the…

Clene is collaborating with APST Research to further explore how CNM-Au8 affects levels of neurofilament light chain (NfL) among amyotrophic lateral sclerosis (ALS) patients being treated in expanded access programs (EAPs). The joint effort is intended to support the accelerated approval of CNM-Au8 for ALS, which Clene intends…