News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to InFlectis BioScience’s experimental therapy IFB-088 for amyotrophic lateral sclerosis (ALS). Orphan drug status is given to treatment candidates with the potential to be safe and effective in rare diseases. In the U.S. rare diseases are defined as…

Regulus Therapeutics, in partnership with researchers at Brigham and Women’s Hospital, has initiated studies to evaluate a library of molecules designed to suppress microRNA-155 (miR-155) — a small molecule that regulates the activity of other genes — as a potential treatment for amyotrophic lateral sclerosis (ALS). The agreement, effective…

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…

A new rapid test is under development that will be able to detect seafood and water levels of an environmental toxin called beta-N-methylamino-L-alanine (BMAA), the exposure to which has been implicated as a risk factor for amyotrophic lateral sclerosis (ALS). The efforts will be spearheaded by Brain Chemistry…

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…

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…

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…