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The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Actimed Therapeutics is developing S-oxprenolol to prevent muscle wasting and body mass loss due to amyotrophic lateral sclerosis (ALS), using proceeds from a recent license agreement with Faraday Pharmaceuticals. Under the agreement, Faraday acquired global rights to develop and commercialize S-oxprenolol for muscle-affecting disorders other than ALS, particularly…

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…

After a protracted campaign to make it happen, Major League Baseball (MLB) agreed to set aside each June 2 to pay tribute to the late Lou Gehrig, the legendary New York Yankees first baseman whose career was cut short by amyotrophic lateral sclerosis (ALS). The inaugural “Lou Gehrig…

People at risk or suspected of having some of the most common neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS), may now access a genetic test at no charge that could diagnose the condition, and open the way to treatment.  Launched by Invitae,…

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…