Targeted therapy may improve function, survival in FUS-ALS

Otsuka Pharmaceutical’s experimental therapy ulefnersen improved a combined measure of function and survival compared with a placebo in people with amyotrophic lateral sclerosis (ALS) caused by mutations in the FUS gene. That’s according to top-line data from the first part of the ongoing, global Phase 3 FUSION clinical…

Ever since my ALS symptoms first appeared, I’ve been trying to rebuild the trust I once had in my body — to trust that my leg will hold me, my foot will clear the floor, and my hand will do what I ask of it. Most of all, I…

Molecular changes linked to inflammation and how brain cells process proteins and genetic material may occur before the onset of amyotrophic lateral sclerosis (ALS) symptoms, a study in a mouse model of SOD1-related ALS suggests. The findings also point to changes in the hypothalamus, a brain region involved in…

My husband crunches on his granola and sips his tea. I watch the rhythmic drip of my liquid “breakfast,” a bag of nutrition tethered to a feeding tube. We share the same couch as always, but the distance between our mornings has grown vast. I flash back to our…

Researchers identified changes in 10 genes that may help diagnose or track amyotrophic lateral sclerosis (ALS), according to a computer analysis of thousands of genes. The study compared whole-blood samples from ALS patients and healthy individuals, uncovering nearly 1,000 genes with altered gene activity, along with related molecular pathways…

Acelot has dosed the first healthy volunteer in a Phase 1a clinical trial testing ACE-2223, its investigational oral treatment candidate designed to target aggregated TDP-43 in people with amyotrophic lateral sclerosis (ALS). In the vast majority of people with ALS, the TDP-43 protein moves to the wrong place in…

Geneuro has received a European patent for GNK-301, its experimental therapy designed to target a viral protein thought to contribute to amyotrophic lateral sclerosis (ALS), and plans to begin Phase 1 clinical trials in the second half of 2027. The antibody-based therapy was developed through a partnership between…

Paradromics’ investigational Connexus brain-computer interface (BCI) enabled a woman with a progressive motor neuron disease to generate her own words in real time, answer open-ended questions, and communicate with her family using synthesized speech. The woman, who had a severe impairment of her ability to speak, was the first…

This morning my husband, Todd, asked me how many boxes of medical gloves we had left, so I took an inventory. We stock medium, large, and extra-large, because different caregivers prefer different sizes. “I think we need to order more large,” I said, “There’s four boxes left.” I paused as…

Mindy Henderson has spent much of her life challenging the limits others have placed on her. When she was diagnosed with spinal muscular atrophy (SMA) — a genetic condition that causes muscles to weaken over time — as an infant, doctors told her parents that she might not live long…