A new study identified a group of sporadic amyotrophic lateral sclerosis (ALS) patients whose disease may arise from a group of “jumping genes” unleashed when TDP-43 protein accumulates in clumps in the brain and spinal cord. These “jumping genes” — which can randomly hop from one location on…
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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
When intrastate rivals University of Washington (UW) and Washington State University (WSU) met Nov. 29 for the storied Boeing Apple Cup Series, the battle wasn’t just about football — it was also about amyotrophic lateral sclerosis (ALS). The teams’ fan bases were vying to see which…
Cromolyn sodium — a U.S. Food and Drug Administration (FDA)-approved anti-inflammatory medication — delayed disease onset and prevented motor neuron degeneration and loss in a mouse model of amyotrophic lateral sclerosis (ALS), researchers report. These neuroprotective effects were accompanied by a decrease in inflammation, highlighting the potential…
Mitsubishi Tanabe Pharma America (MTPA) has enrolled a first patient in a Phase 3 clinical trial of an oral suspension formulation of edaravone in treating amyotrophic lateral sclerosis (ALS). An intravenous form of edaravone (given directly into the bloodstream), developed by Mitsubishi Tanabe Pharma and sold under the brand…
A dissolving oral film form of riluzole, called Exservan and able to be taken by amyotrophic lateral sclerosis (ALS) patients without a need for water, has been approved by the U.S. Food and Drug Administration (FDA). Riluzole was the first ALS treatment, approved by the FDA over two decades…
NurOwn showed a good safety profile, as well as potential efficacy in a Phase 2 clinical trial that included people with rapidly progressing amyotrophic lateral sclerosis (ALS). Trial results, which have been previously reported, have now been published in the journal Neurology in a paper titled, “…
A $106 million Weill Family Foundation initiative will bring together interdisciplinary researchers and clinicians at three West Coast universities in hopes of finding new treatments for brain and nervous system disorders such as amyotrophic lateral sclerosis (ALS). The University of California, Berkeley (Berkeley), the University…
Anelixis Therapeutics has completed its Phase 1 clinical trial testing AT-1501, an investigational antibody, in healthy volunteers and eight amyotrophic lateral sclerosis (ALS) patients. The company also announced it has secured the funds to proceed to a Phase 2 clinical trial of AT-1501. The results of the Phase…
Kadimastem plans to submit an amendment to the protocol of its ongoing Phase 1/2a trial in Israel assessing the use of AstroRx, an “off-the-shelf” stem cell therapy, for the treatment of amyotrophic lateral sclerosis (ALS). The company wants to change the planned dosage in one of the…
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