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…
The ALS Association is urging members of Congress to take action to address issues with instability in leadership at the National Institutes of Health (NIH). At issue is the Trump administration’s decision not to renew the contract of Walter J. Koroshetz, MD, the director of the National Institute of…
Ed and his wife Karen were shocked to hear his ALS diagnosis, but with the help of the VA and others in their community, they discovered they were far from alone. This content is sponsored by Tanabe Pharma America, Inc. (TPA) and is intended for US audiences only. Any other…
Aperture Therapeutics said it is advancing its RNA-targeting therapy APRTX-003 as a potential treatment for amyotrophic lateral sclerosis (ALS). The therapy, currently in preclinical development, is designed to reduce the production of matrix metalloproteinase-9 (MMP-9) by targeting its template RNA molecule. When overactive, this enzyme may lead to…
“I feel like crying,” I wrote in my journal after a second caregiver decided to leave in December. “I can do 18 hours a day on duty, but I need to sleep sometimes.” For a few months, we had had a reliable team of nighttime caregivers for my husband,…
Tanabe Pharma plans to establish a new U.S.-based company to hold the global rights to Radicava and Radicava ORS, two edaravone formulations approved to treat amyotrophic lateral sclerosis (ALS), which Shionogi plans to acquire and operate as a wholly owned subsidiary. “We are pleased to welcome Radicava…
My recent holiday season was overall fun and festive. It did, however, offer a pointed lesson in living with ALS. It began one morning as I was getting ready for a special lunch with relatives. I sat on a folding chair, struggling to put on a sock, and from…
Both people living with amyotrophic lateral sclerosis (ALS) and their caregivers benefit most from flexible, personalized psychological support that combines emotional care and open dialogue, a new study from Germany suggests. Critical, the researchers say, is to create a framework that can be easily adjusted to a patient and…
QRL-101, an experimental oral small molecule being developed by Quralis for amyotrophic lateral sclerosis (ALS), was found in an early clinical trial to engage its intended biological target and reduce nerve cell overactivity in people with ALS. These results come from a proof-of-mechanism Phase 1 clinical study…
