News

Orphazyme announced that it and Worldwide Clinical Trials will continue their collaboration through the long-term extension of a Phase 3 study into arimoclomol, a potential oral treatment for amyotrophic lateral sclerosis (ALS). ORARIALS-01 is a randomized, placebo-controlled and double-blind trial (NCT03491462) underway at 30 centers across North America…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Amyotrophic lateral sclerosis (ALS) progression is associated with higher levels of specific lipids in the spinal cord and changes in their metabolism, a study in a rat model of the disease reports. These findings may represent a defense mechanism against oxidative damage, as well as a potential treatment route for…

A newly developed robotic neck brace may improve quality of life for people with amyotrophic lateral sclerosis (ALS) and allow researchers a more detailed assessment of head and neck movements, as well as disease progression, for people with this condition. The brace was described in a pilot study, titled…

People with rapid weight loss in the earlier stages of amyotophic lateral sclerosis (ALS) — before invasive ventilation is needed to support breathing — are more likely to have a poorer prognosis in its later stages than those who don’t, a study reports. The study, “Body weight variation predicts…

A $3.1 million grant from the National Institute on Aging (NIA) is intended to help two scientists at Northwestern University find new treatments for amyotrophic lateral sclerosis (ALS). The grant was awarded to P. Hande Ozdinler, PhD, a professor of neurology at the university’s Feinberg…

Pharmaceutical company MSD and the Francis Crick Institute are partnering to try to learn more about what causes motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS), so that treatments may be developed. Funded by MSD and the Medical Research Council, the project…

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,…