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.
An experimental oral small molecule being developed by Quralis for amyotrophic lateral sclerosis (ALS) reduced motor neuron excitability as intended in healthy volunteers. That’s according to top-line data from a Phase 1 clinical study (NCT06681441) that tested two doses of QRL-101 against a placebo in healthy adults.
Cognixion is recruiting adults with amyotrophic lateral sclerosis (ALS) for a clinical trial testing ONE Axon-R, a wearable headset that combines augmented reality and brain-computer interface technology with an artificial intelligence (AI) application to facilitate communication. Launched early this year at a single site in the U.S., the…
The ALS Society of Canada (ALS Canada) has named 39 Canadians to be presented the King Charles III Coronation Medal for their efforts into research and advocacy for amyotrophic lateral sclerosis (ALS). The Coronation Medal program, launched in 2023 to mark the coronation of His Majesty King…
Using a smartphone app to self-assess ALS Functional Rating Scale-Revised (ALSFRS-R) scores, a standardized measure of amyotrophic lateral sclerosis (ALS) severity, may be as reliable as clinic-based assessments, a study reports. Its findings suggest this app could be a useful digital tool for remote monitoring of ALS, allowing patients…
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…
Denali Therapeutics‘ oral candidate DNL343 has failed to reduce levels of an established biomarker of nerve cell damage in adults with amyotrophic lateral sclerosis (ALS). That’s according to new data from the DNL343 arm of the HEALEY ALS platform trial (NCT04297683), where patients who received the experimental…
A bipartisan team of lawmakers in the U.S. House of Representatives and Senate is pushing for legislation to help ensure that spouses of veterans who die from amyotrophic lateral sclerosis (ALS) have access to benefits. The Justice for ALS Veterans Act was introduced in the House by Brian Fitzpatrick,…
Pathmaker Neurosystems has received three new patents — two in the U.S. and one in Japan — that cover the use of its noninvasive neuromodulation technology in people with amyotrophic lateral sclerosis (ALS). The patents, which specifically cover the systems and methods used for treating ALS and related diseases,…
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…
