NeuroSense Therapeutics will soon begin a Phase 1 clinical trial to investigate the impact of food on PrimeC, a combo medication being developed as a treatment for amyotrophic lateral sclerosis (ALS), in a group of healthy volunteers. The open-label Phase 1 trial (NCT05232461) is expected to enroll…
Last Sunday, the temperature was in the high 40s, making the tracks in the cross-country ski trail smooth and fast. A gurgling brook carried away snowmelt from a long winter. Birds sang. Two chipmunks scampered up a tree, one chasing the other. Spring skiing is one of my favorite…
A novel system that involves electrodes implanted in the brain allowed a 34-year-old man with amyotrophic lateral sclerosis (ALS) who had completely lost the ability to move voluntarily — known as a “locked-in” state — to communicate with his family again. Using the system, the man was able to…
“To dream the impossible dream, To fight the unbeatable foe, … To reach the unreachable star, … This is my quest.” Vanquish ALS! Last week, my wife and I enjoyed a visit from a dear friend of ours. Interactions with him always have a quixotic effect on my psyche. Just…
Two independent teams of scientists have unraveled the molecular mechanism by which abnormal localization of the TDP-43 protein, which is characteristic of amyotrophic lateral sclerosis (ALS), leads to reduced levels of another protein, called UNC13A, that is important for nerve cell function. The results also show that certain variations…
Amylyx Pharmaceuticals has launched an expanded access program (EAP) in the U.S. that enables eligible adults with amyotrophic lateral sclerosis (ALS) to access the company’s experimental treatment, AMX0035. To be eligible for the program (NCT05286372), patients must have experienced their first signs of weakness three years or…
Mitsubishi Tanabe Pharma Corporation (MTPC) is seeking approval to manufacture and market MT-1186, an oral formulation of edaravone, in Japan for amyotrophic lateral sclerosis (ALS). A similar application recently gained priority review in the U.S., which reduced regulatory review time from the standard 10 months to six months. A…
Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…
Treatment with the cell therapy NurOwn may slow the progression of amyotrophic lateral sclerosis (ALS) for those with a specific variation in the UNC13A gene that is linked with a high risk of the disease. Merit Cudkowicz, MD, chief of neurology at the Massachusetts General Hospital, shared the…
Treatment with Clene Nanomedicine’s experimental medicine CNM-Au8 may decrease the risk of disease progression or death in people with early amyotrophic lateral sclerosis (ALS), according to new analyses from the RESCUE-ALS clinical trial. Data also suggest that the therapy helped to prevent the loss of motor neurons in…
