The Centers for Medicare and Medicaid Services (CMS) has issued a directive requiring that Medicare Advantage plans cover Qalsody (tofersen) to treat people with amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene. The directive comes after the ALS Association worked closely with…

The European Commission has approved Qalsody (tofersen) as a treatment for amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene, known as SOD1-ALS. Qalsody was specifically granted marketing authorization under exceptional circumstances — a pathway recommended when the benefit-to-risk assessment for a therapy is favorable, but…

RAG-17, an investigational therapy from Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status is given to medicines intended to treat life-threatening or chronically debilitating diseases that affect fewer than 200,000 individuals…

A single dose of an artificial microRNA — a tiny RNA molecule that is able to control the activity of certain genes — can lower the activity of SOD1, a gene commonly mutated in patients with familial amyotrophic lateral sclerosis (ALS), a proof-of-concept study shows. According to researchers,…

A team at the Rowan University School of Osteopathic Medicine has received a $1.6 million grant to study the role of the SOD1 protein — whose gene is often mutated in amyotrophic lateral sclerosis (ALS) — in the communication between the gut and the brain. The National Institutes…

A molecule that prevents cell death was able to prevent and reverse clumping of a mutant form of the protein superoxide dismutase 1 (SOD1) — the underlying cause of some familial amyotrophic lateral sclerosis (ALS) cases.  …

A gene editing technology based on CRISPR was able to slow the progression of amyotrophic lateral sclerosis in a mouse model, a new study showed, demonstrating the approach’s potential for gene therapy in people with ALS. The study, “Treatment of a Mouse Model of ALS by…

A recently completed Phase 1 trial studying NPT520-34 — an investigational small molecule being developed by Neuropore Therapies for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS) — has shown the candidate to be safe and well-tolerated in a group of healthy volunteers. The…

AL-S Pharma AG, a biotech company jointly founded and financed by Neurimmune and TVM Capital Life Science, has enrolled the first patient in its Phase 1 clinical trial assessing the safety and tolerability of AP-101, an experimental therapy for amyotrophic lateral sclerosis (ALS). AP-101 is a human…

Mutations causing a complete loss of function of the SOD1 gene lead to severe motor problems, a case study reports, calling for caution in research into silencing this gene as a  potential treatment for people with amyotrophic lateral sclerosis (ALS). The case study, “SOD1 deficiency: a novel syndrome…