News

Mutations in the gene C9orf72, a common genetic cause of amyotrophic lateral sclerosis (ALS), cause immune cells to have an abnormally potent inflammatory response when certain receptors are are activated, a new study suggests. The findings could explain why people with ALS are more likely to develop some autoimmune…

The European Commission has granted orphan drug designation to ILB, Tikomed‘s investigational treatment for amyotrophic lateral sclerosis (ALS). In the EU, this designation is based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP), a part of the European…

The U.S. Food and Drug Administration has granted Prosetin orphan drug designation for the treatment of amyotrophic lateral sclerosis (ALS). Orphan drug designation grants financial incentives to companies developing medications for rare diseases, which might not be profitable enough to pursue without government assistance. While a Phase…

Pharnext’s investigational oral combination therapy PXT864 prevents degeneration of motor neurons and their point of contact with muscle cells, as well as the buildup of toxic TDP-43 protein aggregates — all hallmarks of amyotrophic lateral sclerosis (ALS) — in cellular models of the disease. The data…

Accessing and understanding insurance coverage, and paying for medical treatments and services top the list of stressors that burden people with amyotrophic lateral sclerosis (ALS) in the United States, a survey of patients and caregivers reports. Ten percent of its 444 respondents — in 1 in every 10 —…

A new telehealth app to help people with motor neuron diseases such as amyotrophic lateral sclerosis (ALS) connect with their healthcare providers during the COVID-19 pandemic has been fast-tracked for use in the United Kingdom. The Telehealth in Motor Neurone Disease (TiM) system, developed by the  …

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Two U.K. organizations, the Motor Neurone Disease (MND) Association and Nick Smith Foundation, are partnering on a pilot project to help youngsters affected by motor neurone disease (MND) create memories of their family member or friend. The initiative is being tested in four cities in England: Milton Keynes, Manchester,…

BrainStorm Cell Therapeutics’ cell-based therapy NurOwn increases the number of immunosuppressive cells and lessens inflammation, according to a small preclinical study. These immunoregulatory effects, along with NurOwn’s neuroprotective and neuroreparative properties, may be key to halt the progression of amyotrophic lateral sclerosis (ALS) and other neuroinflammatory diseases, including…

The nonprofit group I AM ALS has opened a global resource to help keep amyotrophic lateral sclerosis (ALS) patients and their caregivers fully aware of clinical research underway. Called ALS Signal: Clinical Research Dashboard and created by patients, caregivers, and advocates, the dashboard offers a user-friendly way to learn about…