NPT520-34 is a new small molecule oral treatment being developed by Neuropore Therapies for potential use against neurodegenerative disorders like amyotrophic lateral sclerosis (ALS) and Parkinson’s disease. The therapy is believed to reduce neuroinflammation in the brain. What is ALS? ALS is a progressive disease…
To help accelerate development of effective treatments for amyotrophic lateral sclerosis (ALS), the ALS Association is investing $3 million in the disease’s first platform trial. The award is $1 million annually for three years to support the platform investigation, a type of clinical study that evaluates the…
A recently completed Phase 1 trial studying NPT520-34 — an investigational small molecule being developed by Neuropore Therapies for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS) — has shown the candidate to be safe and well-tolerated in a group of healthy volunteers. The…
Ultomiris (ravulizumab-cwvz) is an antibody-based medication that was being developed by Alexion Pharmaceuticals as a potential therapy for amyotrophic lateral sclerosis (ALS). However, after a Phase 3 clinical trial indicated that Ultomiris was not effective in treating ALS, Alexion in 2021 discontinued its development for the…
NT0502 is an experimental oral treatment being developed by Neos Therapeutics to reduce excessive salivation — drooling — a common problem in patients with amyotrophic lateral sclerosis (ALS). What is ALS? ALS is a neurological disease characterized by the progressive damage and loss of motor neurons, which are…
In the coming months, Alexion Pharmaceuticals will launch a pivotal Phase 3 clinical trial to test its complement system inhibitor Ultomiris (ravulizumab) in people with amyotrophic lateral sclerosis (ALS) whose motor symptoms began in the prior three years. The CHAMPION-ALS trial — which follows an investigational new…
Including ALS Research Ambassadors — a group of amyotrophic lateral sclerosis (ALS) patients and their caregivers — in discussions about future clinical trial planning and design facilitates changes in study protocol that make the studies more patient-centered. That result is shown in the case of the REFINE-ALS trial, which is…
Kadimastem has finished treating its second group of participants in a Phase 1/2a clinical trial testing the safety and preliminary efficacy of AstroRx, an investigational stem cell therapy for amyotrophic lateral sclerosis (ALS). AstroRx is an off-the-shelf cell therapy consisting…
A newly discovered self-destructive mechanism in mitochondria, the cells’  powerhouses, may be one of the first deficits leading to motor neuron degeneration associated with toxic TDP-43 clumps — a hallmark of amyotrophic lateral sclerosis (ALS). That evidence from a preclinical study suggests available therapies against mitochondrial degeneration might help halt neurodegeneration…
A machine-learning model based on insurance information from amyotrophic lateral sclerosis (ALS) patients may be able to speed diagnosis of the disease in others, research suggests. The findings were presented at the 30th International Symposium on ALS/MND, in the presentation “Machine learning model using insurance…
