News

A viral protein called HERV-K ENV is often detectable in the fluid around the brains of people with amyotrophic lateral sclerosis (ALS), and is toxic to nerve cells, a new study shows. However, blocking this protein with GeNeuro‘s experimental antibody GNK301 reduced those toxic effects in cell…

The first patient has been dosed in an open-label Phase 2 trial that’s evaluating Seelos Therapeutics’ experimental therapy SLS-005 in people with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases associated with the accumulation of toxic protein aggregates. Called a basket trial because it tests one therapy in…

Early treatment with Radicava (edaravone) significantly slows disability progression compared with a six-month delay, including in amyotrophic lateral sclerosis (ALS) patients with poorer lung function, according to a new analysis of a Phase 3 clinical trial. Patients switching from a placebo to Radicava after their lung function fell to…

Early use of Clene Nanomedicine’s experimental therapy CNM-Au8 reduces the risk of death by 70% in people with amyotrophic lateral sclerosis (ALS), compared with patients who experienced a nine-month delay in starting treatment. These are the findings of an updated analysis of the RESCUE-ALS Phase 2 trial…

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…