SOL-257 gene therapy improves outcomes in 2 ALS mouse models

SOL-257, an experimental one-time gene therapy, was well tolerated and significantly improved disease outcomes in two distinct mouse models of amyotrophic lateral sclerosis (ALS). Developed by Sola Biosciences, SOL-257 is designed to clear the toxic TDP-43 protein that accumulates and forms clumps in ALS nerve cells, contributing to…

Last week, after having my yearly mammogram, I got a call from the clinic. The nurse told me that the imaging had revealed a spot that was concerning. I needed to go back for a 3D mammogram and an ultrasound. After I got off the phone, my eyes filled with…

The U.S. Food and Drug Administration (FDA) has signed off on the chemistry, manufacturing, and controls (CMC) clinical development plan for NeuroSense Therapeutics‘ lead candidate for amyotrophic lateral sclerosis (ALS) ahead of a planned Phase 3 study. The Phase 3 trial of PrimeC is due to start…

Pasithea Therapeutics has selected a lead candidate for its PAS-003 program, which is intended to target a “velcro-like” protein to treat amyotrophic lateral sclerosis (ALS). The new therapy is a humanized monoclonal antibody that potently inhibits the alpha-5/beta-1 integrin protein. It was selected after extensive screening and characterization studies…

“Oh my, are you OK? Did you fall? Can I help you up?” Walking into the living room several years ago, my adult daughter was startled to find me on my hands and knees on the floor, huffing and puffing. Twisting to look in her direction, I replied, “Thanks, but…

Two Canadian provinces — Prince Edward Island, and Newfoundland and Labrador — are covering the cost of the oral formulation of Radicava (edaravone) for eligible residents with amyotrophic lateral sclerosis (ALS). Patients will have access through their provincial drug plans to Radicava Oral Suspension, developed along with…

A particular genetic variant that boosts the activity of the NEAT1 gene can push the onset of amyotrophic lateral sclerosis (ALS) forward by more than three years, according to data from a large patient group in China. While the exact function of NEAT1 in ALS needs to be explored,…

When my late husband, Jeff, died of ALS in May 2020, I was awash in grief and lived in a world completely inhabited by memories. I obsessed over our every detail of our lives, large and small. Some thoughts were comforting and reminded me of our happy life together:…

A new advance in technology may pave the way for devices that could allow people with amyotrophic lateral sclerosis (ALS) who have lost the ability to speak to communicate using their thoughts alone. The new tech was described in a study, “High-resolution neural recordings improve the…

I must use the privilege of this column to acknowledge the passing of one of our ALS heroes, Ady Barkan. A young, stalwart, and dedicated husband and father, Ady spent the final few years of his life fighting for those without adequate healthcare in this country. Ady…