Dosing Begins in Phase 3 PHOENIX Trial of AMX0035

Amylyx Pharmaceuticals‘ global Phase 3 clinical trial investigating AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS) has begun dosing participants. The PHOENIX trial (NCT05021536) is expected to include 600 participants whose symptoms began in the past two years, which is a less-stringent criteria than was required for…

When I learned I had amyotrophic lateral sclerosis (ALS), my whole world changed. My husband’s world changed, too, as he suddenly found himself in the role of being my caregiver. For some ALS patients and caregivers, the new responsibilities that follow a diagnosis begin almost immediately. Ours followed a slower…

A small open-label study evaluating the epilepsy medication perampanel in adults with amyotrophic lateral sclerosis (ALS) was halted due to adverse events that affected behavior. Despite the findings, larger clinical trials investigating perampanel in ALS patients are ongoing, which might determine if the medication contributed to behavioral side effects. The…

AC Immune has extended its research collaboration with scientists at the University of Pennsylvania (UPenn) working to uncover how TDP-43 protein clumps contribute to different disease manifestations in amyotrophic lateral sclerosis (ALS). In the partnership’s first two years, researchers with the Perelman School of Medicine at UPenn received…

CNM-Au8, an investigational disease-modifying therapy for people with early amyotrophic lateral sclerosis (ALS), failed to prevent motor neurons loss and lung function decline relative to a placebo, according to top-line data from the RESCUE-ALS Phase 2 clinical trial. The trial, in other words, did not meet its primary…

“How are things going?” a friend asked. I explained that I have been concerned about my husband Todd’s breathing. He’s been waking up with headaches, which could be a sign that his breathing strength has declined due to ALS. He might benefit from noninvasive ventilation, but doesn’t want…

The United to End MND campaign in the United Kingdom is “disappointed” the nation’s government did not accept its plea to invest £50 million (about $68 million) in targeted motor neuron disease (MND) research. MNDs are a group of progressive neurological disorders that includes amyotrophic lateral sclerosis, commonly…

“Well I don’t know why I came here tonight, I’ve got the feeling that something ain’t right. I’m so scared in case I fall off my chair, And I’m wondering how I’ll get down the stairs. Clowns to the left of me! Jokers to the right! Here I am stuck…

Amylyx Pharmaceuticals is seeking approval in the U.S. for AMX0035, its investigational therapy for slowing functional decline in people with amyotrophic lateral sclerosis (ALS). The company submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for regulatory review. The decision had been…

A molecule that blocks the regulatory RNA protein HuR, called SRI-42127, strongly reduces brain inflammation driven by activated glial cells, key players in neurodegenerative diseases like amyotrophic lateral sclerosis, a preclinical study reported. This inhibitor may be of promise in treating ALS and other diseases driven by inflammation in the brain. “Our…