News

For people with amyotrophic lateral sclerosis (ALS) currently on non-invasive ventilation, modifications to existing equipment may help to minimize risk of an infection for both them and their caregivers during the COVID-19 pandemic. The equipment adaptations were discussed in a letter to the editor, “Modification…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

The Phase 2 clinical trial investigating Clene Nanomedicine‘s CNM-Au8 as a potentially disease-modifying therapy for people with amyotrophic lateral sclerosis (ALS) has enrolled more than half of its participants ahead of schedule. RESCUE-ALS (NCT04098406) dosed its first participant in January,…

Mitsubishi Tanabe Pharma America (MTPA) has set up a support program for adults living with amyotrophic lateral sclerosis (ALS) and wanting to learn more about this disease and Radicava (edaravone), an intravenous ALS treatment the company markets in the U.S. Called JourneyMate, the program connects ALS patients…

The U.S. Food and Drug Administration (FDA) has given fast track designation to Orphazyme’s investigational therapy arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS). A therapy candidate is put on the FDA’s fast…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Levels of creatinine kinase — a marker of muscle damage — are elevated in most amyotrophic lateral sclerosis (ALS) patients and appear to be significantly higher among those whose disease progresses at a slower pace, a study in patients and mice shows. These findings suggest that this protein, whose rise…

Changing the composition of bacteria in the gut using antibiotics or fecal transplants may help prevent or reduce the severity of symptoms of amyotrophic lateral sclerosis (ALS), a study in mice with the most common ALS mutation shows. This gut-brain connection may help explain why some patients with this…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

The clear, colorless liquid that fills and surrounds the brain and spinal cord may spread toxic protein aggregates from one nerve cell to others among people with amyotrophic lateral sclerosis (ALS), a study in mice suggests. When repeatedly infused with the cerebrospinal fluid (CSF) from ALS patients, animals…