Note: This story was updated Sept. 21, 2021, to note that 18% of patients, not 80%, experienced increases in the MUNIX(4) sum score at some point during the study. Participants in a placebo-controlled Phase 2 clinical trial of CNM-Au8, Clene Nanomedicine’s investigational oral therapy for amyotrophic…
A research training program in Ireland, called NeuroInsight Fellowship, will use a multi-million grant award to bring established scientists from around the world to that country to advance their skills in neuroscience and data analytics. Two-year fellowships given through this program will allow 33 scientists to further their understanding of…
Clearance was given to open a Phase 1/2 trial evaluating APB-102, a potential gene therapy for amyotrophic lateral sclerosis (ALS) patients who carry mutations in the SOD1 gene, its developer Apic Bio announced. The trial is scheduled to begin later this year or early next year, following U.S. Food and Drug Administration…
A global Phase 3 trial of AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS) intends to enroll up to 600 patients, the therapy’s developer, Amylyx Pharmaceuticals, announced in a press release. Likely to begin in the coming months, the trial — to be called PHOENIX…
Two years after my husband, Todd, was diagnosed with ALS, I read an article that gave me hope. An Israeli rabbi who had ALS saw improvement with NurOwn, an experimental stem cell treatment by BrainStorm Cell Therapeutics. The man went from being in a wheelchair to walking. I…
The European Patent Office will grant MediciNova a patent that covers the combination of MN-166 (ibudilast) and riluzole for the treatment of amyotrophic lateral sclerosis (ALS), MediciNova announced. The patent, once issued, will cover a wide range of doses and dosing regimens for both medications, and it…
SOL-257, an experimental gene therapy developed by SOLA Biosciences, delayed disease progression and prolonged the lifespan of a mouse model of amyotrophic lateral sclerosis (ALS). The gene therapy is designed to reduce the abnormal TDP-43 protein that forms toxic clumps in the cells of ALS patients. “Targeting only…
“I didn’t even know ALS existed until I was told I had it.” That’s what most patients say when telling their ALS story. I said the same thing when asked to share my story at an event in 2010, and I hear the same thing from others today, 11 years…
A Phase 3 clinical trial evaluating Orphazyme’s investigational oral therapy arimoclomol for amyotrophic lateral sclerosis (ALS) has failed to meet its primary and key secondary goals. While no safety issues were reported in the trial (NCT03491462), arimoclomol did not significantly extend patients’ lives nor…
Jan Veldink, a neurologist from the Netherlands whose work in the genetics of amyotrophic lateral sclerosis (ALS) has led to important discoveries about disease risk, won the 2021 Sheila Essey Award, according to a blog post from the ALS Association. The $50,000 award was presented at the recent…
Get regular updates to your inbox.