A $1 million gift from the Manouk Djoukhadjian Family Foundation II will create Quebec’s first philanthropic research chair on amyotrophic lateral sclerosis (ALS). The donation to the Armand-Frappier Foundation will establish the Anna Sforza Djoukhadjian Philanthropic Research Chair to advance ALS research at the Institut national…
Stealth BioTherapeutics announced the initiation of a Phase 1 trial to assess the safety and tolerability of its investigational oral treatment, SBT-272, in healthy volunteers. SBT-272 is being developed to treat neurodegenerative diseases marked by problems in the workings of mitochondria (a cell’s powerhouse), including amyotrophic lateral sclerosis…
The U.S. Food and Drug Administration (FDA) has authorized the evaluation of the first three candidate therapies for amyotrophic lateral sclerosis (ALS) in the HEALEY ALS Platform Trial. This trial model, designed to accelerate the development of ALS therapies by assessing multiple treatment candidates simultaneously, is the first of…
Athletes who played professional football — known as soccer in the U.S. — are more than three times more likely to die of a neurodegenerative disease than are the general population, a study from Scotland has found. Its researchers compared causes of death over 18 years for about 7,700 former professional…
A single spinal injection of an investigational RNA therapy blocked motor neuron degeneration and saved motor function in animal models of familial amyotrophic lateral sclerosis (ALS) linked to SOD1 mutations, a study  reports. Giving the treatment — a virus-delivered gene-silencer targeting the SOD1 gene —  before the onset of…
A recently discovered inflammatory mechanism triggered by Schwann cells —  specialized cells that produce the protective myelin sheath around neurons —  in amyotrophic lateral sclerosis (ALS) can be alleviated with masitinib, a study in rat models shows. The study, “Schwann cells orchestrate peripheral nerve…
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…
