Patricia Inácio, PhD, science writer —

AveXis and REGENXBIO have signed a new licensing agreement to develop therapies for a particular form of ALS and for Rett syndrome. They already have an agreement that covers spinal muscular atrophy therapies. The form of amyotrophic lateral sclerosis that the new agreement covers is the one caused by mutations…

Defects in RNA processing and transport, as well as protein folding, may contribute to amyotrophic lateral sclerosis (ALS) pathology, according to the results of recent research. The study, “Altered nucleocytoplasmic proteome and transcriptome distributions in an in vitro model of amyotrophic lateral sclerosis,” was published in the journal PLOS…

Scientists have found a potential protein biomarker in the cerebrospinal fluid of people carrying the most common mutation linked to amyotrophic lateral sclerosis (ALS) — suggesting that this protein may be useful to test the effects of drugs to treat this form of ALS in future clinical trials. The study, “Poly(GP) proteins are…

A single-case study of a patient with amyotrophic lateral sclerosis (ALS) suggests that long-term invasive motor cortex stimulation via implants of electrodes may slow progression of the disease. The study, “Reduction of disease progression in a patient with amyotrophic lateral sclerosis after several years of epidural motor cortex stimulation” was published…

An analysis of inflammatory marker levels in blood samples from amyotrophic lateral sclerosis (ALS) patients, improves patient categoration and may help design future ALS clinical trials, according to a new study. The study “Systemic inflammatory response and neuromuscular involvement in amyotrophic lateral sclerosis,” was published in the journal…

In a new study, researchers found a protein in the p53 family, called p63, is increased in amyotrophic lateral sclerosis (ALS) and is a significant player in disease progression through the regulation of the activity of genes that promote the breakdown of muscle cells. The study, “Transcriptional activator TAp63 is upregulated in muscular atrophy during…

Researchers revealed that the loss of major histocompatibility complex I (MHCI) is a key step in the development of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease). The study, “Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis,” was published in the journal…

Researchers discovered that a metalloproteinase specialized in the degradation of chondroitin sulfate proteoglycans, called ADAMTS-4, promotes neurodegeneration in a mouse model for human amyotrophic lateral sclerosis (ALS). The study, “ADAMTS-4 promotes neurodegeneration in a mouse model of amyotrophic lateral sclerosis,” was published in the journal Molecular Neurodegeneration. ALS,…

Physical trauma at earlier ages appears associated with an increased risk of amyotrophic lateral sclerosis (ALS), according to a study titled “Physical Trauma and Amyotrophic Lateral Sclerosis: A Population-Based Study Using Danish National Registries” published in the American Journal of Epidemiology. ALS, also known as Lou Gehrig’s disease and Charcot…

Three recent studies published in the journals Nature and Nature Neuroscience identified a mutation in some forms of amyotrophic lateral sclerosis (ALS) and frontotemporal degeneration (FTD) that leads to neuronal death by disrupting the movement of molecules within the cellular nucleus. ALS and FTD are characterized by death…