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The ALS Association is now accepting applications for the Jane Calmes ALS Scholarship Fund, aimed at supporting U.S. students whose lives are affected financially as well as personally by amyotrophic lateral sclerosis (ALS). Going on its third year, the fund helps to cover costs for young people wanting…

Amylyx Pharmaceuticals is planning to file an application by the end of this year requesting that the European Medicines Agency (EMA) approve AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS). This follows a recent announcement that a similar application will be submitted to Health…

Note: This story was updated on April 16, 2021, to note that Scribe’s founding year was 2018, not 2017. Scribe Therapeutics has raised $100 million in funding to develop CRISPR-based gene editing tools and advance its pipeline of therapies for neurodegenerative diseases, such as…

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,…

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…