News

Ab Science’s Potential Treatment for ALS, Masitinib, Named an Orphan Drug by EMA

AB Science announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug status to masitinib, a potential treatment for amyotrophic lateral sclerosis (ALS). Masitinib is an orally administered tyrosine kinase inhibitor targeting mast cells and macrophages, key immune system cells, that works to…

ALS Culprit Protein Chokes Mitochondria, Killing Neurons, According to Recent Discovery

Researchers at Case Western Reserve University School of Medicine have shed light on the mechanism by which a protein called TDP-43 kills nerve cells in amyotrophic lateral sclerosis (ALS) and the related disease frontotemporal dementia. The protein causes neurodegeneration by getting stuck inside mitochondria, preventing these cellular powerhouses from providing energy…

Genetic Splicing Tool Defective in ALS and SMA, Possibly Offering Clues to Treatment

A molecular discovery explaining how motor neuron disease develops draws additional parallels between amyotrophic lateral sclerosis (ALS) and another neurodegenerative disease, spinal muscular atrophy (SMA). The study, titled “Disruption of snRNP biogenesis factors Tgs1 and pICln induces phenotypes that mirror aspects of SMN-Gemins complex perturbation in Drosophila, providing new insights…

Protein Buildup in ALS Brains Linked to Lower Protein Removal Factor

Researchers at Torrey Pines Institute for Molecular Studies, in Florida, discovered that patients with amyotrophic lateral sclerosis (ALS) have lower levels of a factor that controls autophagy, a cell mechanism for protein removal from the brain. The study, “Transcription Factor EB Is Selectively Reduced in the Nuclear Fractions of Alzheimer’s and…

Large ALS Data Analysis Reveals Common Genetic Cause of Disease

Researchers have found that mutations in the NEK1 gene are the most common genetic causes of amyotrophic lateral sclerosis (ALS), accounting for 3 percent of North American and European ALS patients, both sporadic and familial. The study, “NEK1 variants confer susceptibility to amyotrophic lateral sclerosis,” published in…

ALS Patients’ Quality of Life Impacted By Psychological Factors

Researchers at the University Medical Center Utrecht recently studied quality of life among people with amyotrophic lateral sclerosis (ALS) and found that anxiety and depression impact poorer health related quality of life (HRQoL), while higher levels of religiosity and spirituality are associated with better overall (global) quality of life (QoL). The research paper,…