FDA Accepts Pharma’s New Drug Application for ALS Therapy Edaravone

The U.S. U.S. Food and Drug Administration (FDA) has accepted Mitsubishi Tanabe Pharma’s new drug application (NDA) for edaravone as a treatment for amyotrophic lateral sclerosis (ALS). A decision regarding the intravenous treatment is expected by June 16, 2017, based on the U.S. Prescription Drug User Fee Act. If it is…

University of Miami researchers found high concentrations of toxins associated with neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), in the fins and muscles of 10 different shark species, some of them threatened by extinction due to overfishing. Such findings suggest that restriction of shark meat and fins consumption may have important…

Researchers at Harvard Medical School have made an important breakthrough in amyotrophic lateral sclerosis (ALS) research, identifying an enzyme that plays a key role in the demise of nerve cells in ALS patients and paving the way for research in new therapies. Their discovery has already set in motion the…

Cytokinetics recently announced that patient enrollment for its VITALITY-ALS clinical trial is now complete. The study, designed to assess the effects of tirasemtiv, a novel skeletal muscle activator, on respiratory function in ALS patients has enrolled over 700 people at 81 centers in 11 countries. VITALITY-ALS (NCT02496767) is an international, randomized, double-blind and placebo-controlled trial in…

Proteasome, a cellular component, plays a key role in the maintenance of proper levels of proteins inside cells. Two researchers at the Massachusetts General Hospital (MGH) have identified, through studies in a roundworm, key molecules in the pathway through which cells sense and repair proteasome abnormalities. Such findings may lead to new…

Oxford BioDynamics is expanding its program for biomarker discovery with tests for amyotrophic lateral sclerosis (ALS) in a clinical study in Malaysia, using its EpiSwitch technology. Researchers hope the new study, one of the largest ALS studies in Asia to date, will allow the development of a diagnostic test that…

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…