News

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…

ABBV-CLS-7262, an experimental compound being developed by Calico Life Sciences and AbbVie for amyotrophic lateral sclerosis (ALS), will likely serve as the sixth therapy in the multi-regimen HEALEY ALS platform trial. HEALEY (NCT04297683) is testing the potential of several ALS treatment candidates simultaneously with a goal of speeding…

A new €2.5 million (about $2.5 million) grant from the European Union (EU) will help advance a prototype vaccine for amyotrophic lateral sclerosis (ALS) associated with mutations in the C9orf72 gene — its most common genetic cause. The European Innovation Council EIC Transition Grant will be used to further develop the vaccine,…

PandaOmics, an artificial intelligence (AI) platform that looks at vast amounts of data to find “druggable” molecules, has identified more than two dozen new targets with therapeutic potential for amyotrophic lateral sclerosis (ALS), according to a recent study. Target discovery is a first step in the path to…

Amyotrophic lateral sclerosis (ALS) type 4 — a juvenile and slowly progressive form of the neurological disease, called ALS4 — is driven by abnormal mechanisms in both the central nervous system and the immune system, a new study reports. In particular, patients with this condition have increased levels of inflammatory…

An approved medication for heart-related chest pain reduced the frequency and severity of muscle cramps in people with amyotrophic lateral sclerosis (ALS) in a small pilot study. Called ranolazine, the medication was found to be safe and generally well-tolerated — the clinical trial’s key endpoints, or goals. Gastrointestinal…

An imaging analysis platform that examines the brain’s white matter in MRI scans could help to diagnose amyotrophic lateral sclerosis (ALS), a new study suggests. The results were presented by Braintale at the European Academy of Neurology (EAN 2022) Congress, held both digitally and in Vienna, Austria. The poster…

Corestem is still enrolling participants in its Phase 3 clinical trial of NeuroNata-R (lenzumestrocel), a stem cell therapy that is conditionally approved to treat amyotrophic lateral sclerosis (ALS) in South Korea. The trial, ALSummit (NCT04745299), is evaluating the safety of repeat NeuroNata-R injections and how well…

A U.S. Food and Drug Administration (FDA) advisory committee will meet  Sept. 7 to discuss the approval of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS), Amylyx Pharmaceuticals announced. While the application was granted priority review earlier this year, with a final decision expected by June…

Using the approved treatment Radicava (edaravone) in a real-world setting resulted in a similar safety profile as that reported in clinical trials, with no new safety signals identified, according to a new report. The analysis involved more than 5,000 people with amyotrophic lateral sclerosis (ALS) who received the…