A gene therapy designed to block the activity of SARM1 protein prevented the loss of axons ­­­­— long projections that connect nerve cells and transport information ­­­­— making the therapy a potential strategy to reduce the loss of peripheral nerves in several neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), according to a mouse…

Amyotrophic lateral sclerosis (ALS) patients who complete the ongoing Phase 3 REFALS clinical trial evaluating levosimendan (also known as ODM-109) will soon have the opportunity to enroll in an open-label extension study. Levosimendan is an oral treatment developed by Orion that is being investigated for its ability to relieve breathing…

A small molecule known as mir-494-3p might play a protective role in the survival of motor nerve cells, a discovery that could lead to the development of new therapies for amyotrophic lateral sclerosis (ALS), a study suggests. The study, “Micro-RNAs secreted through astrocyte-derived extracellular vesicles cause neuronal network…

The loss of a key enzyme, called adenosine deaminase, in astrocytes ­­­­— the energy-supporting cells of neurons ­­­­— leads to a toxic accumulation of molecules that contributes to the death of motor neurons seen in amyotrophic lateral sclerosis (ALS), a study shows. The study, “Astrocyte adenosine deaminase loss increases…

Suppressing an enzyme known as MAP4K4 extended the survival of motor neurons collected from mice and patients with amyotrophic lateral sclerosis (ALS), and decreased the accumulation of toxic proteins associated with the disease, a study reports. These findings from the study, titled “MAP4K4 Activation Mediates Motor…

I have ALS. That awareness has prompted a series of rapid-fire questions. While many of us would describe existence as being comprised of multiple chapters, I now have two distinctly different lives. Prior to ALS, I had led an unremarkable life largely characterized by good fortune. Some of…

Do you speak with an “ALS accent”? I know I do. Fortunately, my family and close friends have learned to translate my slow, slurred words into meaningful conversation. However, to the Alexa device that lives in our back bedroom and the Echo Show that sits near me, my ALS…

An immune response triggered by T-cells contributed to the elimination of motor neurons in a mouse model of amyotrophic lateral sclerosis (ALS), a study shows. The study, “Cytotoxic CD8+ T lymphocytes expressing ALS-causing SOD1 mutant selectively trigger death of spinal motoneurons,” was published in PNAS. ALS is…

SIRION Biotech and Denali Therapeutics have signed a new licensing agreement to develop a new generation of adeno-associated virus (AAV) vectors, harmless viruses used as vehicles to deliver gene therapies, to allow therapies to reach into the central nervous system (CNS) of people  with amyotrophic lateral sclerosis (ALS). The…

An upcoming webinar will focus on the novel approach to clinical trial design in the Modifying Immune Response and Outcomes in ALS (MIROCALS) trial, which aims to break the current impasse in therapy development for amyotrophic lateral sclerosis (ALS). During the webinar, P. Nigel Leigh, a motor neuron disease…