News

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,…

ProMIS Neurosciences announced plans to advance a monoclonal antibody against toxic TDP-43 protein clumps as a potential treatment for amyotrophic lateral sclerosis (ALS). The candidate antibody therapy, called PMN267, has shown promise in preclinical experiments conducted at independent institutions, the company also reported in a press release. Nearly all cases…

Treatment with AMX0035 significantly extended the median survival of amyotrophic lateral sclerosis (ALS) patients by more than 10 months compared with a placebo, a new analysis of the Phase 2/3 CENTAUR clinical trial indicates. That survival difference was even greater in patients who continued to receive AMX0035 in the open-label…

WVE-004, Wave Life Sciences‘ experimental therapy for people with amyotrophic lateral sclerosis (ALS) associated with mutations in the C9ORF72 gene, has demonstrated proof-of-concept efficacy in cell and animal models, a new study shows. The nucleic acid-based therapy was able to “potently” reduce the toxic RNA molecules and small proteins…

Merit E. Cudkowicz, MD,  is the winner of the inaugural Target ALS Rebecca Luker Courage Award in recognition of her work and efforts to help  amyotrophic lateral sclerosis (ALS) patients and the ALS community. Cudkowicz is the chief of neurology at Massachusetts General Hospital, a professor…

More than 5,000 people are diagnosed with amyotrophic lateral sclerosis (ALS) each year, and the out-of-pocket costs of care are an estimated $250,000 for each person with the disease, according to the ALS Association. These are just a couple of the statistics about ALS that advocates, organizations, and…

Treatment with Coya Therapeutics‘ ALS001, a regulatory T-cell (Treg) therapy being developed for amyotrophic lateral sclerosis (ALS), was found to reduce markers of oxidative stress — a type of cell damage — and inflammation in a small early trial. The experimental Treg therapy was tested in a Phase 1…