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…

Researchers at the Czech Academy of Sciences report that two mutations, previously linked to amyotrophic lateral sclerosis (ALS), disrupt the ability of a calcium channel to transport calcium ions in nerve cells. The study, titled  “CACNA1H missense mutations associated with amyotrophic lateral sclerosis alterCav3.2 T-type calcium channel…

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…

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…

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…

The ALS Association and the biopharmaceutical company Cytokinetics have announced the renewal and expansion of their partnership, established last year, to better treat amyotrophic lateral sclerosis (ALS) patients and educate the public about the disease. The renewal includes the continuation of Cytokinetics’ Gold Level Sponsorship of the National Walks…

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…

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,…

How cells recycle waste, an essential process to cellular health known to go astray in amyotrophic lateral sclerosis (ALS) and other diseases, is more complex and interrelated than previously thought, researchers at the Neuromed Institute in Italy report. Their study, “The Autophagoproteasome a Novel Cell Clearing Organelle in Baseline and…

ProMIS Neurosciences announced the start of a research program to identify new therapeutic targets on neurotoxic strains of a protein, TDP43, implicated in the development of  amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). TDP43 (TAR DNA-binding protein-43) is present in every cell and plays a key role in the response to oxidative stress, an…