Assessing the Contents of the Toolbox While in Exile
“By the rivers of Babylon Where we sat down And there we wept When we remembered Zion But the wicked carried us away in captivity Required from us a song How can we sing King Alpha song In…
“By the rivers of Babylon Where we sat down And there we wept When we remembered Zion But the wicked carried us away in captivity Required from us a song How can we sing King Alpha song In…
Lately, I’ve been pondering the many ways our lives have changed for the sake of safety, like tamper-proof packaging, airport screening, and two-step password verification. I get it. Identity thieves are everywhere, so are scammers, along with the just plain crazies. We might be a little safer, but I…
Cells contain certain chaperone proteins that can break down the protein clumps found in amyotrophic lateral sclerosis (ALS) and Huntington’s disease, but don’t always activate the right proteins at the right time, a recent study shows. “[The cells] do not always realize there is a problem, or know…
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…
“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…
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…