Statins, often prescribed to lower cholesterol levels, are not associated with poorer overall survival in people with amyotrophic lateral sclerosis (ALS) and should not be discontinued on that basis, a German study has found. The study, “Statins, diabetes mellitus, and prognosis of Amyotrophic…
News
Two bills making their way through Congress are intended to improve access to medical therapies for those most in need, and in particular, those with amyotrophic lateral sclerosis (ALS). Sen. Mike Braun, R-Ind., announced his plan to introduce a bill to hasten the regulatory process for making promising therapies…
The COVID-19 pandemic has imposed new challenges on diagnosing, monitoring, and caring for people with amyotrophic lateral sclerosis (ALS), from the absence of in-person visits to a lack of available equipment at hospitals. But the expansion of telemedicine may be a way to overcome these challenges, and allow…
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…
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