Patricia Inácio, PhD, science writer —

Boston-based Flex Pharma has announced positive topline results from its exploratory Phase 2 trial testing investigational therapy FLX-787 in Australian amyotrophic lateral sclerosis (ALS) patients with frequent muscle cramps. The data showed that FLX-787 significantly reduced the intensity of cramp-associated pain and stiffness compared to placebo. The positive results support the…

Findings from a Phase 3 study of tirasemtiv to improve muscle strength — particularly of respiratory muscles — in amyotrophic lateral sclerosis (ALS) patients will likely be announced for a first time on Dec. 8 at the 28th ALS/MND meeting in Boston, a vice president of Cytokinetics, its developer, said…

Researchers at Germany’s University of Würzburg have discovered how a malfunctioning autophagy – a cell’s natural self-degradation and cleanup system – plays a central role in the development of amyotrophic lateral sclerosis (ALS) and other motor neuron diseases. The study, “Plekhg5-regulated autophagy of synaptic vesicles reveals a pathogenic mechanism…

Voyager Therapeutics has recently presented data of its leading adeno-associated virus (AAV) gene therapy for several neurological diseases, including amyotrophic lateral sclerosis (ALS), at the Congress of the European Society of Gene and Cell Therapy (ESGCT), Oct. 17-20 in Berlin, Germany. In their poster, “Translation of Intrathecal Delivery of an AAV Gene…

Scientists have discovered how adult skin cells can be converted into motor neurons without transitioning them through a stem cell state. The findings promise to help researchers better understand motor neuron diseases, such as amyotrophic lateral sclerosis (ALS). The study, “MicroRNAs Induce a Permissive Chromatin Environment that…

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…