Margarida Maia, PhD, science writer —

ANX005, an experimental medication being developed by Annexon Biosciences for neurodegenerative and autoimmune diseases, resulted in a slowing of disease progression in adults with amyotrophic lateral sclerosis (ALS). That’s according to preliminary data from eight patients taking part in a Phase 2a clinical study (NCT04569435), whose main…

Masitinib, an oral medicine developed by AB Science, has won orphan drug status for treating amyotrophic lateral sclerosis (ALS) in Switzerland. The status is granted by the Swiss Agency for Therapeutic Products (Swissmedic) to therapies that seek to treat life-threatening or chronic diseases affecting no more than…

A low dose of aldesleukin, an immunotherapy approved for certain cancers, slowed disease progression and significantly improved survival in people with amyotrophic lateral sclerosis (ALS), particularly in those with less aggressive disease. These are the most recent results from the MIROCALS Phase 2 trial (NCT03039673), which tested aldesleukin…

DNL343, an investigational oral small molecule developed by Denali Therapeutics, can extensively enter the brain and reduce the cellular stress response that contributes to amyotrophic lateral sclerosis (ALS) progression. That’s according to an interim analysis of data from a Phase 1b clinical trial (NCT05006352), in which DNL343 was…

Neuvivo’s NP001 (sodium chlorite) may help slow the progression of amyotrophic lateral sclerosis (ALS) by taming the systemic inflammation caused by gut bacteria leaking into the bloodstream, according to a new analysis of Phase 2 trial data. The findings point to microbial translocation, which occurs when bacteria break through…

EpicentRx has been awarded funding from FightMND to study how well RRx-001, its lead small molecule, may protect nerve cells against damage caused by amyotrophic lateral sclerosis (ALS), a disease also known as motor neuron disease (MND). “We are very pleased to receive this support from FightMND…

The first patients have been dosed in a Phase 1/2a clinical study that’s testing AMDXP-2011P for its ability to trace disease-associated toxic protein clumps in the eyes of adults with amyotrophic lateral sclerosis (ALS) and Parkinson’s disease, which may allow earlier diagnosis. The study, dubbed PROBE (NCT05542576),…

Using a digital tool to assess disease severity remotely is feasible for people with amyotrophic lateral sclerosis (ALS) and may improve patient monitoring between visits to the clinic, a study suggests. The tool makes the revised ALS Functional Rating Scale (ALSFRS-R) available to patients via a computer or mobile…

Stem cells engineered to mature into nerve-supporting cells that produce GDNF — a key factor for motor neuron survival — were safely delivered into the spinal cord of people with amyotrophic lateral sclerosis (ALS), according to data from a Phase 1/2a clinical trial. These cells, dubbed CNS10-NPC-GDNF, were…

People with amyotrophic lateral sclerosis (ALS) have significantly higher blood levels of lead, a toxic heavy metal, than those without the disease, a review study found. While the findings strengthen accumulating evidence of a link between lead exposure and the risk of developing ALS, they do not definitively prove…