News

One-time delivery of a CRISPR-based gene therapy significantly reduced the buildup of toxic RNA molecules resulting from C9ORF72 mutations in cells and mouse models of amyotrophic lateral sclerosis (ALS), the therapy’s developer, Locanabio, reported. The approach was designed using the company’s CORRECTx platform, which uses CRISPR technology to…

After several years of support, the late-stage biopharmaceutical company Cytokinetics is reaffirming its commitment to the ALS Association and its efforts to battle amyotrophic lateral sclerosis (ALS) and assist the ALS community. In addition to backing nationwide organizational efforts, Cytokinetics has, for more than a decade, provided support…

The estimated 2017 U.S. prevalence of individuals with amyotrophic lateral sclerosis (ALS) was 24,821, or 7.7 per 100,000 people, according to a recent report from the National ALS Registry. While a statistical model was used to account for potentially missing data, the research team emphasized that this number is…

Treatment with AMX0035 significantly prolonged the time that people with amyotrophic lateral sclerosis (ALS) in the CENTAUR clinical trial did not require permanent ventilation or tracheostomy by over seven months, a new study shows. “People living with ALS often require medical interventions like mechanical ventilation as the disease…

Team Gleason and Synchron have entered into a partnership aiming to advance a technology that offers greater accessibility for people with amyotrophic lateral sclerosis (ALS) and other conditions that affect physical movement. Synchron is developing brain computer interfaces, or BCIs — devices that can detect activity in…

A research team has developed a new way to search for genes and gene mutations that may contribute to the development of rare diseases, bypassing some of the challenges typically associated with genetic analyses. For diseases like amyotrophic lateral sclerosis (ALS) in which genetic factors are thought to…

The U.S. Food and Drug Administration (FDA) has approved Mitsubishi Tanabe Pharma America (MTPA)’s oral suspension formulation of edaravone, Radicava ORS, for the treatment of amyotrophic lateral sclerosis (ALS). The oral therapy is designed to offer the same efficacy as MTPA’s Radicava (edaravone) — an FDA-approved, into-the-vein therapy…

A partnership between Rolls Royce and the U.K.-based Motor Neurone Disease (MND) Association is helping people with amyotrophic lateral sclerosis (ALS) preserve the sound of their voices simply by reading a book into a computer. Called voice banking, the process is a way to record a person’s voice…

Silence ALS, a new initiative supported by Target ALS, set as its goals discovering and developing personalized antisense oligonucleotide (ASO) treatments for amyotrophic lateral sclerosis (ALS) patients with rare disease-causing mutations, while advancing understanding of the disease’s overall biology. The initiative, a collaboration between the n-Lorem Foundation, which…

A Phase 2 clinical trial has begun dosing patients to evaluate the safety and effectiveness of Denali Therapeutics‘ investigational oral therapy SAR443820 in people with amyotrophic lateral sclerosis (ALS). The study, dubbed HIMALAYA (NCT05237284), is recruiting about 260 adults at sites in the U.S., Belgium, France, Germany,…