First Project Mosaic initiative: Developing patient-specific models

New study IDs better method to detect gene defects in sporadic ALS

Long-read DNA sequencing is a more accurate method than short-read sequencing for detecting certain defects in genes associated with an increased risk of sporadic amyotrophic lateral sclerosis (ALS), a new study suggests. A relatively new technique, long-read sequencing can capture data on thousands to hundreds of thousands of nucleotides,…

New study may explain why ALS therapy BIIB078 didn’t work

The investigational antisense oligonucleotide (ASO) therapy BIIB078 may have failed in amyotrophic lateral sclerosis (ALS) clinical trials because it wasn’t adequately reversing key disease processes in the brain and spinal cord. Analyses of body fluids and tissue from participants showed that, while BIIB078 reached key tissues, it couldn’t entirely…

While living with ALS, panic leads to stress again and again

A friend who stopped by for a visit last weekend asked my husband, Todd, “Anything new?” “Not really,” he said. “So no more choking incidents?” she asked. “There have been choking incidents, and Kristin has to clear my lungs every couple days,” he replied, “but you asked if there was…

AP-101 holds promise for some with ALS, topline data show

AP-101, AL-S Pharma’s amyotrophic lateral sclerosis (ALS) therapy, was safe and well tolerated and led to clinically meaningful benefits for breathing and survival after one year of treatment. That’s according to topline data from a now-complete Phase 2a study (NCT05039099) that tested the therapy in 73 adults with…

The pleasures of finding humor in my life with ALS

“I should be an ALS comedian. I know I could, too — that is, if it weren’t for my darn ALS.” That’s what I tell myself on the days when life’s funny moments come at me fast and furious. It’s another example of how this disease keeps on challenging…

5 students from ALS families in Canada win scholarships

Five students from across Canada whose lives have been directly affected by amyotrophic lateral sclerosis (ALS) received scholarships to help them pursue post-secondary education. Each ALS Canada Kevin Daly Bursary recipient will receive $2,500 for the 2025–2026 academic year. The scholarship program is designed for students with a parent,…

Trial in Australia, Europe will test heart drug in ALS patients

A clinical trial in Australia and Europe will test whether trimetazidine, a medication commonly used to treat heart conditions, could help improve function and quality of life for amyotrophic lateral sclerosis (ALS) patients. The trial, led by University of Queensland researcher Shyuan Ngo, PhD, and her team at the…