HEALEY ALS platform trial launches new arm testing oral therapy NUZ-001

Dosing has begun in a new arm of the HEALEY ALS platform trial, with Regimen I — the ninth — testing NUZ-001, an oral small molecule developed by Neurizon Therapeutics to slow declines in function and extend survival in people with amyotrophic lateral sclerosis (ALS). As a platform…

Next week, researchers, clinicians, industry leaders, and families will gather at the 2026 MDA Clinical & Scientific Conference, hosted by the Muscular Dystrophy Association (MDA), to discuss the latest advances in neuromuscular disease research and care. Ahead of the event, Bionews, the parent company of this site, sat…

Blood levels of lactate — a molecule involved in how the body produces energy — may help predict survival and short-term weight changes in people with amyotrophic lateral sclerosis (ALS), according to a new study from Australia and Japan. Researchers found that patients with lower blood lactate levels had…

AB Science has identified a new blood biomarker, produced by certain immune cells, that may help assess the activity of its experimental therapy masitinib in people with amyotrophic lateral sclerosis (ALS). The biomarker, which the company did not disclose for patent protection reasons, is capable of identifying patients…

The phrase “bucket list,” which has become popular in today’s culture, refers to a set of experiences or activities a person hopes to accomplish before dying. These might include visiting the Grand Canyon and skydiving, for example. While it was further popularized by the 2007 movie of the same name,…

Wake me up if Todd is in distress,” I said to his nighttime caregiver. “Of course,” she replied. It’s not something I typically say to one of my husband’s regular caregivers, but I felt on edge. I said good night and tried to get some rest, but my mind was…

The experimental therapy QRL-201 successfully reached its biological target and showed early signs that it may slow disease progression in people with sporadic amyotrophic lateral sclerosis (ALS), according to new Phase 1/2 trial data. These interim results from the proof-of-concept ANQUR trial (NCT05633459) suggest the treatment can…

I have to confess: I envy people who multitask. It is something I used to do with ease, but ALS has firmly put those days in the rearview mirror. It’s not even that I am “tasking” much these days; I find myself simply reminiscing about the times when I…

The Muscular Dystrophy Association (MDA) has launched its annual MDA Shamrocks campaign, described as the nation’s largest St. Patrick’s Day-themed fundraiser, to support people living with amyotrophic lateral sclerosis (ALS) and other rare neuromuscular diseases. During the campaign, held throughout February and March, thousands of retailers across the…