News

German researchers have identified a new gene — called NEK1 — that appears to be responsible for familial amyotrophic lateral sclerosis (ALS). Loss of function mutations in the gene were more common in ALS patients than in healthy controls, according to findings published in the journal Brain. Familial ALS…

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…

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…

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…

New research by MIT Sloan Prof. Andrew W. Lo, Dana-Farber Cancer Institute‘s David Weinstock, M.D., and Vahid Montazerhodjat, an MIT post-doctoral fellow, offers a solution for the excessive costs associated with breakthrough therapies that already exist for certain diseases such as ALS: securitized consumer healthcare loans (HCLs). The research introducing this practical…