Nine patients with an ultra-rare form of amyotrophic lateral sclerosis (ALS) caused by mutations in the CHCHD10 gene were treated with an experimental antisense oligonucleotide (ASO) that’s designed to silence the disease-causing mutations. The treatment was developed as part of Silence ALS, an initiative that designs ASOs, that is,…

The U.S. Food and Drug Administration (FDA) has authorized Sineugene Therapeutics to begin a Phase 1/2a trial of its experimental gene therapy, SNUG01, in people with amyotrophic lateral sclerosis (ALS). The global trial will assess the treatment’s safety, tolerability, and preliminary efficacy through a dose escalation and expansion…

People living in California can help fund amyotrophic lateral sclerosis (ALS) research when filing their state taxes. The California ALS Research Network Voluntary Tax Contribution Fund allows Californians to contribute to research projects selected by the ALS Network’s scientific advisory committee. At least $250,000 must be raised…

The weeks immediately following my late husband’s ALS diagnosis were the scariest and most unsettling of my life. Even with Jeff’s physical symptoms pointing toward ALS, nothing could’ve prepared us to actually hear those words when they were delivered in a windowless examination room in Baltimore, in November 2018.

NUZ-001, which was previously known as monepantel, slowed lung function declines by nearly 50% in people with amyotrophic lateral sclerosis (ALS), according to new analyses from a Phase 1 trial. This slowing of respiratory declines was strongly correlated with slower declines in overall function that were observed in…