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…
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…
Get regular updates to your inbox.
