Dosing of healthy adults begins in Phase 1 study of oral CVN293

A first healthy volunteer has been dosed in a Phase 1 clinical study evaluating how safe and well tolerated is CVN293, a treatment candidate for amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease, the therapy’s developer, Cerevance, announced. The treatment, given orally, is designed to block a protein…

The European Medicines Agency (EMA) is now expected to issue a decision in the first months of 2024 on the conditional approval of masitinib as an add-on oral therapy for amyotrophic lateral sclerosis (ALS). The deadline extension follows a request, seeking more time, from AB Science, the therapy’s developer,…

“Find your own Calcutta. Find the sick, the suffering, and the lonely, right where you are — in your own homes and in your own families, in your workplaces and in your schools. You can find Calcutta all over the world, if you have eyes to see.” — Mother…

Disaster preparedness, including stockpiling goods, owning and knowing how to use emergency equipment, and being aware of community resources, is lacking among amyotrophic lateral sclerosis (ALS) patients and caregivers, a recent Japanese study suggests. While patients who needed a ventilator were generally found to be better prepared during power…

People with amyotrophic lateral sclerosis (ALS) are not at a higher risk of complications due to a spinal tap than are healthy individuals or those with other neuromuscular disorders, according to a small U.S. study. Findings show that ALS patients have a relatively lower risk for such adverse events,…

Every person with amyotrophic lateral sclerosis (ALS) should be offered genetic testing to check for the most common ALS-causing mutations, according to a new set of expert guidelines. “These evidence-based, consensus guidelines will support all stakeholders in the ALS community in navigating benefits and challenges of genetic testing,”…

PathMaker Neurosystems has launched a Phase 1 clinical trial that will test its experimental noninvasive nerve-modulating device in people with amyotrophic lateral sclerosis (ALS). This is the first time that the device — called the MyoRegulator and designed to slow disease progression in ALS — will…

Treatment with Ultomiris (ravulizumab-cwvz) — a therapy approved for several rare diseases that works by blocking activation of part of the immune system called the complement cascade — failed to slow the progression of amyotrophic lateral sclerosis (ALS) in a Phase 3 clinical trial that had been launched…

Last week, I set my husband, Todd, up on his computer before heading out for my weekly ukulele group. It’s difficult to leave my husband for long, because he’s paralyzed by his ALS. But my class lasts only an hour and I’d be less than 10 minutes away. In…

Inhibiting the NLRP3 inflammasome — an immune system protein complex believed to contribute to the inflammation that drives amyotrophic lateral sclerosis (ALS) — alone was not sufficient to ease spinal cord inflammation in a mouse model of the disease, according to recent research. NLRP3 is overactive in ALS and…