News

People with mutations in the C9ORF72 gene who develop amyotrophic lateral sclerosis (ALS) show signs of damage to certain brain regions years before the appearance of disease symptoms, a study reports. The findings suggest that looking for changes in brain structures could help predict which people with C9ORF72 mutations…

Widetrial, an integrated service and technology platform, is coming on board to support an expanded access program (EAP) that will provide the investigational therapy MN-166 (ibudilast) to amyotrophic lateral sclerosis (ALS) patients who aren’t eligible for clinical trials. The program is funded under a $22 million federal…

Eli Lilly will develop therapies against two drug targets for amyotrophic lateral sclerosis (ALS) that were identified and validated by Verge Genomics as part of a three-year collaboration between the companies. The targets, which weren’t disclosed, were discovered with Converge, Verge’s platform that draws on human…

RAG-21, a therapy Ractigen Therapeutics is developing for amyotrophic lateral sclerosis (ALS) linked to mutations in the FUS gene, has been awarded orphan drug status by the U.S. Food and Drug Administration (FDA). Orphan drug status in the U.S. is granted to products aiming to prevent, diagnose, or treat…

The ALS Association has awarded a “groundbreaking” grant to Tiziana Life Sciences to support a small, early-stage clinical trial testing foralumab nasal spray for amyotrophic lateral sclerosis (ALS). The upcoming trial intends to investigate the safety and effectiveness of two foralumab doses in 20 ALS patients. It…

Answer ALS and Cedars-Sinai have made openly available their amyotrophic lateral sclerosis (ALS) patient-based stem cell and biodata repository to researchers worldwide. The largest of its kind, the repository contains biological and clinical data from nearly 1,000 ALS patients, as well as specialized nerve cells called induced pluripotent…

A newly awarded grant from the ALS Association will support Asha Therapeutics‘ work to advance ASHA-624, a small molecule being developed for amyotrophic lateral sclerosis (ALS), into a first-in-human clinical trial that’s expected to begin early next year. The grant was through the Lawrence and Isabel Barnett…

The European Medicines Agency (EMA) has recommended that monepantel, now named NUZ-001, be granted orphan medicinal product designation to treat amyotrophic lateral sclerosis (ALS). The positive opinion will be reviewed by the European Commission, which is expected to issue a final decision in December. If granted, the designation will provide…

A Phase 2a clinical trial testing Rho kinase (ROCK) inhibitor Bravyl (oral fasudil) in people with amyotrophic lateral sclerosis (ALS) has completed patient enrollment for its high-dose group, the company said. While the REAL trial (NTC05218668) was initially slated to only test a 180 mg daily dose of…

A new biopharmaceutical company has launched in the U.S. with more than $100 million in financing and a goal to develop a genomic medicine targeting the UNC13A protein as a novel treatment for people with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Trace Neuroscience kicked off its…