PrimeC is safe and well-tolerated for the treatment of amyotrophic lateral sclerosis (ALS) and shows potential to slow disease progression and lung function decline, according to interim results from a Phase 2a clinical trial. Given these results, developer NeuroSense Therapeutics will continue investigating PrimeC with additional analysis…

Newly developed compounds derived from ebselen could be used to treat some types of amyotrophic lateral sclerosis (ALS), a recent study suggests. The study, “Novel Selenium-based compounds with therapeutic potential for SOD1-linked amyotrophic lateral sclerosis,” was published in EBioMedicine. About a fifth of familial…

The ALS Association and I AM ALS have opened a petition calling for the earliest possible approval of Amylyx’s experimental oral treatment AMX0035, after a Phase 2/3 trial found the therapy to be safe and to slow functional decline in amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease. Addressing the…

Younger cycad seeds can contain more toxins than older seeds, and their widespread consumption by people on occupied Guam during World War II could be the environmental origin of the rare sporadic amyotrophic lateral sclerosis (ALS) cluster found there, according to a recent study. Guam residents emerged from their wartime…

A Phase 1 clinical trial of a gene-based therapy for amyotrophic lateral sclerosis (ALS) is now recruiting patients in one of its European centers. The Clinical Research Facility at St James’s Hospital Dublin, in Ireland, has enrolled its first patient and is one of four European centers to participate…

Amylyx’s experimental oral treatment AMX0035 safely and effectively slows functional decline in amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease, according to full data from the CENTAUR Phase 2/3 trial. Only changes in upper limb strength were significantly different from those seen in placebo-treated patients, the data show. Meanwhile,…

The Phase 3 clinical trial testing an oral suspension formulation of edaravone for amyotrophic lateral sclerosis (ALS) has resumed dosing and patient recruitment, its developer, Mitsubishi Tanabe Pharma America (MTPA), announced. The trial (NCT04165824) was launched in November, but was paused in March due to the COVID-19 pandemic. The study,…

Mutations in the gene C9orf72, a common genetic cause of amyotrophic lateral sclerosis (ALS), cause immune cells to have an abnormally potent inflammatory response when certain receptors are are activated, a new study suggests. The findings could explain why people with ALS are more likely to develop some autoimmune…

The European Commission has granted orphan drug designation to ILB, Tikomed‘s investigational treatment for amyotrophic lateral sclerosis (ALS). In the EU, this designation is based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP), a part of the European…

The U.S. Food and Drug Administration has granted Prosetin orphan drug designation for the treatment of amyotrophic lateral sclerosis (ALS). Orphan drug designation grants financial incentives to companies developing medications for rare diseases, which might not be profitable enough to pursue without government assistance. While a Phase…