Dropping the levels of microRNA-218 (miR-218) — a small molecule that regulates the activity of other genes — below a certain threshold leads to severe motor neuron damage, paralysis, and death in a mouse model of amyotrophic lateral sclerosis (ALS), a study shows. The findings, combined with the fact that…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
A national agency in Norway has given AB Science the go-ahead to resume enrollment in AB19001, a Phase 3 clinical trial investigating the safety and efficacy of the company’s investigational oral therapy masitinib in people with amyotrophic lateral sclerosis (ALS). AB had voluntarily suspended recruitment and treatment…
Mast cells, a type of immune cell, infiltrate the spinal cord of amyotrophic lateral sclerosis (ALS) patients and rodent models of the disease, exhibiting pro-inflammatory features that are likely involved in motor neuron and microvasculature damage, a study suggests. Notably, treating these mice models with masitinib — an experimental oral…
Eledon Pharmaceuticals announced that its Phase 2 study of AT-1501 in people with amyotrophic lateral sclerosis (ALS) should be fully enrolled by year’s end. Top-line trial results are expected between April and September 2022. “Enrollment in our ALS study is progressing well and we anticipate completing enrollment in…
I AM ALS, a nonprofit dedicated to helping people affected by amyotrophic lateral sclerosis (ALS), is expanding its efforts in scientific research and public policy. The organization’s new Science and Policy program will include three key focuses, according to a press release emailed to ALS News Today.
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
A Temple University research team received a National Institutes of Health (NIH) grant totaling $1.25 million to explore the molecular underpinnings of neuronal degeneration in amyotrophic lateral sclerosis (ALS) and other diseases. With this five-year grant, the team led by Gareth Thomas, PhD, an associate professor of neural sciences…
Alexion Pharmaceuticals is stopping further work on Ultomiris (ravulizumab) — its approved therapy for complement-associated rare blood disorders — as a potential treatment of amyotrophic lateral sclerosis (ALS) based on interim data from the CHAMPION-ALS Phase 3 clinical trial. While top-line results were expected by mid-2022, the study’s independent data…
People affected by amyotrophic lateral sclerosis (ALS) have come up with plenty of creative ways to raise money for research and care — whether it’s the ALS Ice Bucket Challenge, re-creating Olympic events, or Major League Baseball’s designation of June 2 as Lou Gehrig Day. Joe…
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