‘Mission Impossible’ Merits a Blockbuster Cast

“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…

I felt twinges of grief as I shopped for a card for a young couple’s wedding. First card: “Wishes on this special day for happiness, joy, and love, along with a future that will bring everything you are dreaming of.” Sigh. I wish my husband and I had a future.

“And you may find yourself living in a shotgun shack … And you may ask yourself, ‘Well, how did I get here?’ … Letting the days go by, same as it ever was.” Although I’m not proud of it, my life, pre-ALS, was largely a fortunate stream of autopilot idleness.

An ambitious Irish research project, called Precision ALS, will combine clinical research, data science, and artificial intelligence (AI) to identify the multiple — and potentially targetable — factors involved in the development and progression of amyotrophic lateral sclerosis (ALS). The goal is to use this information to determine which treatments…