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…
The grief that accompanies ALS is multifaceted. I grieve my husband’s loss of function and the loss of our dreams. Just when we find a new normal, there is further decline. I let my grief out on the pages of my journal. I cry, I scream in the car when…
Dosing of healthy volunteers has been completed in a Phase 1 clinical trial evaluating Neos Therapeutics‘ experimental therapy NT0502 for chronic sialorrhea (excessive drooling), a common problem in people with amyotrophic lateral sclerosis (ALS). The open-label study includes 30 healthy adults who were assigned randomly to take one of…
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…
The new year arrived bearing good and bad news. The good news is that 2020 marks my 10th year of living with ALS and I’m still here! The bad news is that I still have ALS. And, gosh darn, I thought we’d have a cure by now. But we…
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…
Amyotrophic lateral sclerosis (ALS) patients receiving the lowest dose of Kadimastem’s cell therapy candidate, AstroRx, experienced a significant reduction in disease progression in the three or four months after treatment, updated findings from the company’s Phase 1/2 clinical trial show. In subsequent…
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…
Like many families, we played games over the holidays. Because my husband, Todd, is completely paralyzed due to ALS, we chose games in which he could participate. Apples to Apples worked well for him. I set his cards in the game’s box top, and he told me which card to…
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…
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