News

Maze Therapeutics is advancing the development of a lead gene therapy candidate for amyotrophic lateral sclerosis (ALS), which works by suppressing the activity of a potent genetic modifier called ATXN2. Genetic modifiers are genes or genetic variants that can increase or reduce the severity of a condition without necessarily…

Ionis Pharmaceuticals is opening a Phase 3 safety and efficacy trial of ION363 (jacifusen) in amyotrophic lateral sclerosis (ALS) patients with confirmed mutations in the FUS gene, a known cause of juvenile-onset disease. The trial (NCT04768972) will enroll up to 64 people, ranging in age from 12 to 65,…

The National Institutes of Health (NIH) has awarded a $450,000 grant to a university research team to explore mutations in the FUS gene that contribute to amyotrophic lateral sclerosis (ALS), and to study genes showing a potential…

A higher than expected proportion of amyotrophic lateral sclerosis (ALS) patients — most without a family history of ALS — had mutations in genes associated with the disease, according to a study in 100 patients in the U.K. “Our study found that 42 per cent of patients involved in…

The Muscular Dystrophy Association (MDA) is urging people with amyotrophic lateral sclerosis (ALS) to take part in a new study that’s seeking to track disease progression through a mobile app. In addition to ALS, the ongoing study is enrolling adults with primary lateral sclerosis (PLS) or related neuron…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Note: This story was updated April 6, 2021, to note that the study is expected to conclude late this summer, instead of 2024. The ALS Association, Muscular Dystrophy Association (MDA), and ALS Finding a Cure have given an additional $1.1 million to support work on a cell therapy…

Biogen will not offer early access to tofersen, its experimental therapy for familial amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, at least until data from its pivotal clinical trial are analyzed. Now in its Phase 3 portion, the study (NCT02623699) is testing the…

Retrotope has dosed the first patients in its recently-initiated Phase 2 clinical trial evaluating RT001 for the treatment of amyotrophic lateral sclerosis (ALS). Enrollment is open across four European sites for patients ages 20–75 whose disease symptoms began less than three years ago and do not need…

Roche Canada’s Artificial Intelligence Centre of Excellence (AI CoE), in collaboration with Answer ALS and EverythingALS, opened a challenge that asks the global artificial intelligence (AI) community to model solutions for key questions in amyotrophic lateral sclerosis (ALS). Called End ALS Challenge, the digital competition also…