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…
The ALS Association, a U.S.-based nonprofit whose mission is to help find a cure for amyotrophic lateral sclerosis (ALS), has issued a statement strongly opposing plans for cuts in National Institutes of Health (NIH) funding, saying any such reductions would obstruct progress in ALS research. The organization also…
Having access to results from prognostic tests — including neurofilament light chain (NfL) and the ALS Functional Rating Scale-Revised (ALSFRS-R) — is informational, personally relevant, and relevant to research for people with amyotrophic lateral sclerosis (ALS), a study suggests. The perceived burden related to receiving the tests results was…
AB Science will launch a new Phase 3 clinical trial to test its investigational oral therapy masitinib in people with amyotrophic lateral sclerosis (ALS) whose disease is progressing normally and who haven’t completely lost physical function, according to a company update. The trial, called AB23005, will replace…
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