The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NPT520-34, an investigational anti-neuroinflammatory molecule, for the treatment of amyotrophic lateral sclerosis (ALS), Neuropore Therapies announced. “We are very pleased with receiving this orphan drug designation from the FDA’s Office of Orphan Product Development for NPT520-34…
News
Motor Neurons in Familial ALS Have Mutation-Specific Alterations in Glutamate Signaling, Study Found
Stem cell-derived motor neurons from people with familial amyotrophic lateral sclerosis (ALS) have mutation-specific alterations in glutamate receptors and calcium signals, which may alter nerve signaling and play a role in the disease, a study found. These findings may lead to future…
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…
Clusters containing a protein called LAT use specific adapters to move and drive the activation of T-cells to fight off infection, according to a study, the findings of which may help design immune cells with more selective effects, the researchers suggest. The study, “A composition-dependent molecular clutch…
Scientists have created a new type of split-hand index — which identifies the loss of the pincer grasp — that is able to distinguish people with amyotrophic lateral sclerosis (ALS) from healthy individuals at the earliest stages of the disease. Results from the study, “Split-hand index in…
Biopharma AC Immune has entered a research collaboration with scientists at the Perelman School of Medicine at the University of Pennsylvania (UPenn) to uncover why TDP-43 misshapes and accumulates in the nervous system of some people, like those with amyotrophic lateral sclerosis (ALS).
People in China with amyotrophic lateral sclerosis (ALS) can now access edaravone (brand names Radicava, Radicut, among others), following its approval by the National Medical Products Administration, the country’s regulatory agency. The infusion therapy (delivered into the veins) is meant to slow ALS progression and will…
Complete Loss of SOD1 Function Leads to Severe Motor Problems, Study Says in Warning to Researchers
Mutations causing a complete loss of function of the SOD1 gene lead to severe motor problems, a case study reports, calling for caution in research into silencing this gene as a potential treatment for people with amyotrophic lateral sclerosis (ALS). The case study, “SOD1 deficiency: a novel syndrome…
People with amyotrophic lateral sclerosis (ALS) have an altered composition of their gut microbial community, with an increase in harmful microbes and a decrease in beneficial microorganisms, according to a new small study. This altered gut microbiota could drive digestive problems in those with ALS, the researchers said. The…
Underweight ALS Patients at Risk for Poorer Outcomes at Later Stages of Disease, Study Suggests
Being underweight when they start supported feeding puts patients with amyotrophic lateral sclerosis (ALS) at risk for poor outcomes at later stages of the disease, an Italian study has found. The study, “Nutritional prognostic factors for survival in amyotrophic lateral sclerosis patients undergone percutaneous endoscopic gastrostomy placement,”…
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