News

Changes in neurofilament light chain (NfL) levels — a biomarker of nerve cell damage — were deemed by an advisory committee reasonably likely to predict clinical efficacy from tofersen in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The unanimous vote from the Peripheral and Central Nervous…

A potential new genetic risk factor for amyotrophic lateral sclerosis (ALS) has been discovered by researchers at the University of Washington. The scientists say a mutation in which a region of the WDR7 gene is repeated more times than usual may increase the risk of ALS. “We believe that…

Four people with amyotrophic lateral sclerosis (ALS) each experienced a marked slowing in disease progression with COYA 302, an experimental immune-modulating therapy tested in a small proof-of-concept clinical trial, according to Coya Therapeutics, its developer. Based on these findings, Coya is planning to start work on further trials to…

Amyotrophic lateral sclerosis (ALS) patients treated with Radicava (edaravone) tend to go longer before reaching disease milestones such as needing a walking aid or breathing support, according to an analysis of insurance data. The findings were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in…

Amylyx Pharmaceuticals is facing a new hurdle — a second round of questions from a European Medicines Agency (EMA) committee — in its quest to have AMX0035 approved as a treatment for amyotrophic lateral sclerosis (ALS) in Europe. The questions, from the Committee for Medicinal Products for…

Anew Medical‘s targeted gene therapy candidate, ANEW-202, eased multiple mechanisms associated with amyotrophic lateral sclerosis (ALS), leading to improved muscle function and survival in a mouse model of the disease. The gene therapy, licensed on an exclusive worldwide basis from the Autonomous University of Barcelona, in Spain, is…

QurAlis has secured $88 million to advance the clinical development of QRL-101 and QRL-201, its lead candidates for amyotrophic lateral sclerosis (ALS). The new series B financing, a second round of funding after the company met certain research milestones, brings the total investment to $143.5 million. The proceeds will…

Radicava ORS, an oral formulation of Radicava (edaravone), is now available to U.S. veterans living with amyotrophic lateral sclerosis (ALS), the therapy’s developer, Mitsubishi Tanabe Pharma America (MTPA), announced. The therapy was added to the U.S. Department of Veterans Affairs National Formulary (VANF) in June 2022, shortly after its…

The Muscular Dystrophy Association (MDA) is hosting its 16th annual MDA Atlanta Night of Hope Gala: A Benefit for ALS Research to raise funds for amyotrophic lateral sclerosis (ALS) research and care. The event will be held 6-11 p.m. EDT March 25 at the InterContinental Buckhead Atlanta. It…

Renowned neurologist and amyotrophic lateral sclerosis (ALS) investigator Merit Cudkowicz, MD, of Massachusetts General Hospital has been named this year’s recipient of the Muscular Dystrophy Association (MDA) Legacy Award for Achievement in Clinical Research. The award, which recognizes outstanding accomplishments in neuromuscular research, will be presented March 20…