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…
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,…
I love learning some new skills, but not all of them. Thirteen years ago, I took a creative writing class that set me off on a journey that has included three novels, a children’s book, and now a weekly column. This week, I hope to take a…
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…
Glaciers are known for their slow movement and the transformative change that they leave in their wake. Most move only a few centimeters a day. Yet they produce lakes, cliffs, moraines, valleys, mountain arêtes and horns, and pronounced landscape striations. Perhaps stem cell research…
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…
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…
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