News

Abnormal Proteins Found in ALS and Like Diseases Are ‘Tagged’ to Form Clumps, Study Reports

Scripps Research Institute (TSRI) scientists have identified a new player in the ribosome-associated quality control complex that “tags” abnormal proteins, prompting them to aggregate and form clumps. This study, for a first time, shows that proteins can be marked for aggregation, and may constitute a new molecular pathway underlying neurodegenerative diseases such…

ALS Study Finds Higher Than Usual Levels of Axon ‘Repellent’ Protein in Patients’ Motor Cortex

Motor neurodegeneration in amyotrophic lateral sclerosis (ALS) can begin in axons at neuromuscular junctions and later spread to the cell body in the spinal cord, in a phenomenon known as the “dying back” hypothesis. Proteins involved in axonal regeneration may, for this reason, represent a potential therapeutic target in ALS. A research team from Germany found…

Biohaven’s ALS Drug Candidate BHV-0223 Receives Positive FDA Feedback

Portage Biotech and Biohaven Pharmaceuticals recently announced they have received positive feedback from the U.S. Food and Drug Administration (FDA) on their Pre-Investigational New Drug Application (PIND) interaction regarding Biohaven’s investigative drug BHV-0223, intended for treatment of amyotrophic lateral sclerosis (ALS). BHV-0223 is the glutamate modulating compound riluzole, contained in the tablet…

ALS Drug Developer Cytokinetics Joins Rare Disease Day Efforts to Educate and Promote Change

Cytokinetics announced that it has joined with efforts by the European Organisation for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD) to raise awareness for Rare Disease Day. Cytokinetics is focused on discovering, developing, and commercializing new types of muscle activators, and has developed the amyotrophic lateral sclerosis (ALS) drug candidate…