New ALS study suggests targeting VCP could protect nerve cells

Overactivity of a molecular system that normally helps clear unwanted proteins may contribute to nerve cell damage in certain genetic forms of amyotrophic lateral sclerosis (ALS), a new study suggests. The findings may open new avenues for treatment, researchers wrote in the study titled “Aberrant nuclear…

Mindwalk Holdings has identified and validated antibodies that selectively target an abnormal form of the TDP-43 protein, which is found in about 97% of amyotrophic lateral sclerosis (ALS) cases and is also linked to other neurodegenerative diseases. The candidates include antibodies that could potentially be given as a…

The first few months after my husband, Craig, died from ALS in April 2022 were incredibly hard and a bit of a blur. I went through the motions, not really knowing or caring what I did. Once the relatives had gone, the funeral was over, and the house was…

When my late husband, Jeff, was diagnosed with ALS in 2018, I knew it would bring emotional challenges for both of us. I expected — correctly, as it turns out — that we’d feel scared, anxious, and sad. I recognized the immediate and lingering shock it brought in different…

The first patient has been dosed in a Phase 2 clinical study testing RAG-17, a therapy being developed by Ractigen Therapeutics for people with amyotrophic lateral sclerosis (ALS) who carry mutations in the SOD1 gene (SOD1-ALS). The progression into the Phase 2 portion of the trial follows the…

Every step of an ALS journey is difficult, but somehow we’re wired with the ability to forget much of the pain and remember the good. I wrote last week about our current challenge of finding nighttime caregivers. After getting all of the shifts covered, one of our caregivers…

Clene is scheduled to meet with the U.S. Food and Drug Administration (FDA) in the coming months to discuss new data and a potential path toward accelerated approval for CNM-Au8, an oral therapy being developed for amyotrophic lateral sclerosis (ALS). The company plans to argue that the…

I’m always on the lookout for strategies to help my day-to-day life with ALS go more smoothly. Recently, I found a surprising spark of inspiration in a junk drawer in the kitchen. It wasn’t something hiding inside the drawer that helped; instead, it was my frustrating encounter with the…

The first participants have been dosed in an early Phase 1 clinical study that’s testing NRG5051, a first-in-class treatment candidate for amyotrophic lateral sclerosis (ALS) and Parkinson’s disease, in healthy volunteers. Developed by NRG Therapeutics, the experimental therapy is designed to restore the health of mitochondria, known…

Note: This story has been updated Jan. 16, 2026, to correct that once the therapy is delivered, the gene, not the viral vector, remains inside the cell. The U.S. Food and Drug Administration (FDA) has granted fast-track designation to VectorY Therapeutics’ lead investigational therapy, VTx-002, to treat amyotrophic…