Supporters will celebrate the 5th annual Lou Gehrig Day on June 2. The yearly event honors the legacy of the Major League Baseball (MLB) Hall of Famer whose high-profile struggle with amyotrophic lateral sclerosis (ALS) brought widespread attention to the rare neurodegenerative disease. Through awareness and fundraising initiatives,…
Clene Nanomedicine is on track to seek accelerated approval of CNM-Au8 for treating amyotrophic lateral sclerosis (ALS) by the end of the year, backed by neurofilament light chain (NfL) biomarker data from its expanded access program (EAP). Those analyses, which are intended to address a recommendation…
The Muscular Dystrophy Association (MDA) will celebrate the 25th anniversary on June 25 of its Wings Over Wall Street Gala, an annual fundraiser to support research for amyotrophic lateral sclerosis (ALS). The event will take place at the Edison Ballroom in New York City, starting at 6…
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…
