News

Cytokinetics Inc. has presented its Vision 2020: Empowering Our Future – an initiative designed to expand the company’s pipeline until 2020, as well as advance its muscle biology-directed drug candidates toward late-stage development and marketing strategies, to tackle the unmet medical needs of people living with impaired muscle…

The ratio of N-acetyl aspartate (NAA) to glutamate is related to disease duration in patients with amyotrophic lateral sclerosis (ALS), a finding that supports the notion of glutamate toxicity as a contributing factor in ALS pathology. Earlier studies have indicated that the neurotransmitter glutamate is involved in ALS. People with sporadic ALS…

A panel of leading amyotrophic lateral sclerosis (ALS) researchers recently published a review study, supported by the ALS Association, detailing the state of disease biomarker development and calling for the formation of an ALS Biomarker Consortium to speed up research into more effective disease treatments. The review, titled “ALS biomarkers…

Motor neuron degeneration in amyotrophic lateral sclerosis (ALS) patients is caused by several factors, including inflammation and oxidative stress. Uncovering the multiple pathways leading to ALS may suggest new ways to intervene therapeutically. University of Pittsburgh researchers found the Receptor for Advanced Glycation End Products (RAGE) and its ligands, or…

A report in the journal JAMA Neurology revealed positive findings from the first Phase 1/2 clinical trial and the Phase 2 dose escalation study investigating BrainStorm’s NurOwn for the treatment of amyotrophic lateral sclerosis (ALS). NurOwn is a stem cell technology employing adult cells isolated from the disease-affected patient, developed…

Researchers at the National University of Singapore have suggested there is a fine balance between amyotrophic lateral sclerosis (ALS)-related protein TDP-43’s normal function and its potential to cause neurodegeneration. The degeneration seems to occur due to pathological factors that exaggerate the protein’s membrane association, resulting in the loss of its physiological functions as well as…

University of Bern researchers have discovered that the synaptic protein Homer-3 is linked to the death of Purkinje motor neurons in Spinocerebellar ataxia type 1 (SCA1), a motor neuron disease that, like amyotrophic lateral sclerosis (ALS), is characterized by extensive motor neuron loss. The study, titled “Impaired mTORC1-Dependent Expression of…

A new proof-of-concept study reports converting adult skin cells from ALS patients into muscle neurons without first transforming them into stem cells. The patient-derived muscle neurons were dysfunctional, but when treated with a small molecule, the team could restore neuronal function. The use of induced pluripotent stem cells (iPSCs)…

A research group — coordinated by Giuseppe Legname from The Laboratory of Prion Disease, SISSA, Trieste, Italy — has found a way to build artificial prions, assembling the proteins in a serial manner. The study was performed in collaboration with the Carlo Besta Neurological Institute in Milan and the results were published in the journal…

Researchers at the Salk Institute have discovered that a set of messenger RNAs, controlled by the microRNA miR-218, define a specific network of neuronal genes that are repressed to prevent neuromuscular damage and neurodegeneration. Loss of miR-218 was seen to lead to the onset of neurodegenerative diseases such as amyotrophic lateral…