News

A Phase 2 and potentially pivotal study investigating pegcetacoplan (APL-2) as a potential treatment for amyotrophic lateral sclerosis (ALS) is now enrolling patients, Apellis Pharmaceuticals, the therapy’s developer, announced in a press release. The MERIDIAN trial (NCT04579666) is enrolling up to 228 adults with sporadic ALS and…

BrainStorm Cell Therapeutics is sharing the details of the design of its Phase 3 clinical trial evaluating NurOwn as a treatment for people with rapidly progressing amyotrophic lateral sclerosis (ALS), showing that the trial is…

Libra Therapeutics has officially announced its launch with a platform dedicated to developing new treatments for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The company also has secured $29 million in Series A funding. The funds will be used to expand Libra’s team…

Scribe Therapeutics and Biogen, a biotechnology company focused on neurological diseases, have established a research partnership to develop new gene therapies for amyotrophic lateral sclerosis (ALS) using the gene-editing technology CRISPR-Cas9. The collaboration will focus initially on the development and commercialization of CRISPR-based therapies that address some…

The Muscular Dystrophy Association (MDA) has awarded five new grants totaling more than $1.6 million to boost research of amyotrophic lateral sclerosis (ALS). These grants add to the $168 million already donated to ALS research by…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

Target ALS is teaming up with Spartan on a global initiative to raise funds for research that will contribute to the fight against amyotrophic lateral sclerosis (ALS). Called “The Toughest Challenge for the Hardest Fight,” the inaugural campaign asks participants to raise funds by participating in…

Saphyr, an ultra-long DNA analysis technology by Bionano Genomics, is the first to successfully measure very large repeat expansions in the C9ORF72 gene, the underlying cause of some amyotrophic lateral sclerosis (ALS) cases, the…

Creatine kinase, a marker of muscle damage, is significantly elevated in people with amyotrophic lateral sclerosis (ALS), and higher levels of this protein correlate with better survival rates in patients, a recent study found. These findings suggest that monitoring creatine kinase levels is useful for predicting disease progression…

A combined analysis using people of Japanese, Chinese, and European ancestry identified five new genes associated with the non-familial, sporadic form of amyotrophic lateral sclerosis (ALS).  These findings further an understanding of the genetic basis of sporadic ALS, and may support…