MDA 2023: Designer DNA drug for ALS shows promise in mouse studies

In most cases of amyotrophic lateral sclerosis (ALS), dysfunction of a protein known as TDP-43 leads to abnormally low levels of another protein, stathmin-2, which is sufficient to drive nerve cell dysfunction, new data suggest. In an aim to restore stathmin-2 levels, researchers created a designer DNA treatment. Now,…

“So tell us, Dagmar, what’s your latest project?” The question came from my neurologist as we chatted during my recent ALS clinic visit. She asked not only for herself, but for the medical student who was also in the room. As I prattled on, part of my brain reflected…

On average, a person with amyotrophic lateral sclerosis (ALS) will require a wheelchair to get around within two years of the initial onset of disease symptoms. Findings underscore the condition’s rapid advance, and the need for new ALS treatments that work to slow disease progression, its researchers noted.

On a bitterly cold morning in New York last weekend, I woke before the sun and walked through the empty city streets to the Times Square subway station. From there, I caught a train to Brooklyn, the starting line for the United Airlines NYC Half, where more than…

Changes in neurofilament light chain (NfL) levels — a biomarker of nerve cell damage — were deemed by an advisory committee reasonably likely to predict clinical efficacy from tofersen in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The unanimous vote from the Peripheral and Central Nervous…

My husband’s nose wouldn’t quit bleeding this past Sunday morning, preventing him from using his noninvasive ventilator. Todd is paralyzed from ALS, so he sat in his power wheelchair in front of the bathroom sink as I went to work. I packed his nostril with wadded up…

A potential new genetic risk factor for amyotrophic lateral sclerosis (ALS) has been discovered by researchers at the University of Washington. The scientists say a mutation in which a region of the WDR7 gene is repeated more times than usual may increase the risk of ALS. “We believe that…

Four people with amyotrophic lateral sclerosis (ALS) each experienced a marked slowing in disease progression with COYA 302, an experimental immune-modulating therapy tested in a small proof-of-concept clinical trial, according to Coya Therapeutics, its developer. Based on these findings, Coya is planning to start work on further trials to…

ALS has taught me many life lessons, and a very important one is to never give up. For example, some days I get up, get going, and suddenly notice that one of my body parts isn’t moving as well as the day before. It’s a small, quirky thing, like…

Amyotrophic lateral sclerosis (ALS) patients treated with Radicava (edaravone) tend to go longer before reaching disease milestones such as needing a walking aid or breathing support, according to an analysis of insurance data. The findings were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in…