Gene Seen to Protect Motor Neurons May Serve as ALS and SMA Therapy, Study Suggests

The protein synaptotagmin 13 (SYT13) protected motor neurons from degeneration in cell and animal models of amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), a study reports, suggesting a gene therapy based on this protein’s related gene, SYT13, would treat these and other diseases of motor nerve cells.

“Lately it occurs to me what a long, strange trip it’s been.” –Grateful Dead, “Truckin’“ Last month, I quietly acknowledged the 13th anniversary of my ALS diagnosis. As in years past, I mentioned the occasion’s passing to no one. Instead, the…

Impaired processing and buildup of protein aggregates associated with amyotrophic lateral sclerosis (ALS) are linked to a protein that regulates the uptake of substances into the cell, a process known as endocytosis, a study found. Enhancing endocytosis may be an…

People with amyotrophic lateral sclerosis (ALS) may benefit from at-home monitoring using an app, a study found. The study, “Telehealth as part of specialized ALS care: feasibility and user experiences with ‘ALS home-monitoring and coaching’,” was published in Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. Providing…

Antibodies that target proteins wrongly expressed in the brain and driven by mutations in C9ORF72, a cause of amyotrophic lateral sclerosis (ALS), eased symptoms and prolonged survival in a mouse disease model, a new study shows. Its researchers suggest that such antibodies could be used to treat people with…

Cognitive impairment in amyotrophic lateral sclerosis (ALS) may be linked to the buildup of the protein TDP-43 in the brain, a new study suggests. However, TDP-43 alone likely isn’t the cause of such impairment, and due to the study’s small sample size, more research is needed to clarify…

I’m not the same person I was 10 years ago, before my husband, Todd, was diagnosed with ALS. The brutal reality of the disease has changed me in three ways: 1. I swear more I’d never done much swearing. When I was 15, I had just gotten my driver’s permit…

The biotechnological company INmune Bio has been awarded a $500,000 grant from the ALS Association to further develop a therapy that might reprogram the innate immune system in people with amyotrophic lateral sclerosis…

PrimeC, a new combination treatment by NeuroSense Therapeutics to slow down or halt the progression of amyotrophic lateral sclerosis (ALS), has received orphan drug status from the U.S. Food and Drug Administration (FDA), and is being investigated in two recently-initiated clinical trials. The decision to…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…