News

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…

Scientists have long linked abnormal versions of the protein TDP-43 to neurodegenerative diseases such as ALS, but they haven’t had a full understanding of how it causes nerve cell deterioration. A Japanese study in mice indicates that elevated levels of the normal version of TDP-43 can play a role in…

The Muscular Dystrophy Association (MDA) has awarded an MDA Venture Philanthropy (MVP) grant worth $233,200 to Iron Horse Diagnostics to support the development of a biomarker test for amyotrophic lateral sclerosis in the United States. The prognostic test, which was released in Europe in June under a license agreement…

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…

Biohaven Pharmaceutical has dosed the first amyotrophic lateral sclerosis (ALS) patient with its sublingual formulation of riluzole, BHV-0223, in a pivotal Phase 2/3 trial. The trial aims to demonstrate that BHV-0223’s effects are at least equivalent to Rilutek, an approved tablet formulation of riluzole. The trial initiation was supported by…

Option Care Enterprises, a Chicago-based independent provider of home and alternate treatment site infusion services, has partnered with the ALS Association to offer Radicava (edaravone) treatment from the comfort of their homes. To date, more than 100 amyotrophic lateral sclerosis (ALS) patients are already receiving Radicava — the first…

The U.S. Food and Drug Administration (FDA) has given the green light to Biohaven Pharmaceuticals to advance sublingual BHV-0223, which regulates glutamate communication between neurons, into clinical trials for amyotrophic lateral sclerosis (ALS) patients. The FDA’s  permission to go ahead with the study followed Biohaven’s submission of an investigational new drug…

Repeated severe trauma to the body or mild injuries to the head — especially at midlife — raise a person’s risk of ALS, a European case-control study from the EURALS consortium shows. The new research, “Trauma and amyotrophic lateral sclerosis: a european population-based case-control study from…

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…