uniQure is planning to start enrolling patients in a second dose group as part of a clinical trial testing AMT-162, its one-time gene therapy for amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The open-label Phase 1/2 study, called EPISOD1 (NCT06100276) is intended to…
Neuropeutics has partnered with LifeArc to advance work on a small molecule therapy against TDP-43 clumping as a potential disease-modifying treatment for amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND). Studies in cellular and animal models found the candidate treatment,…
It takes longer for people with amyotrophic lateral sclerosis (ALS) who are being treated with Radicava (edaravone) to reach certain disease progression milestones, such as using a walking aid or a wheelchair, compared with those not treated with the medication. A study of real-world data also indicates the…
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…
Moments of daily life with my husband Todd’s ALS frequently put both of us in panic mode. It makes me wonder if we should take more precautions — but there are trade-offs. The other day, for example, food got stuck in Todd’s throat while I was feeding him chicken…
Ncardia has launched a stem cell-derived model of human microglia to advance research into neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS), where these cells play an important role. Microglia, the resident immune cells in the brain, help support the function of nerve cells, but if they become overly…
Last week, I fully intended to write a new “Living Well With ALS” column. I sat down, flipped open my laptop, gazed out the window, and got lost in a stare. As the minutes ticked by, my computer screen finally clicked off, my mind blanked out, and I had…
A machine learning analysis of gene activity in spinal cord samples from people with amyotrophic lateral sclerosis (ALS) revealed new genes associated with the neurodegenerative disorder, a study reports. The newly identified genes included those involved in the function of energy-producing mitochondria, lipid (fat) and iron metabolism, and the…
New York received the highest mark on the ALS Association’s latest report cards, which grade states based on how effectively they’re using public policy to serve the amyotrophic lateral sclerosis (ALS) community. In the state policy report cards, New York was the only state to earn…
NP001 (sodium chlorite) was associated with a significant survival benefit among a subset of amyotrophic lateral sclerosis (ALS) patients who did not show signs of disease progression when treated with the investigational therapy in Phase 2 clinical trials, according to new data from the two studies. In this group…
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