The U.S. Food and Drug Administration has agreed to expand Tiglutik, a liquid oral form of riluzole, to include giving it via a feeding tube to treat amyotrophic lateral sclerosis (ALS), ITF Pharma, the company marketing Tiglutik, announced. The approval of administering the therapy via percutaneous endoscopic…
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MediciNova‘s experimental therapeutic ibudilast (MN-166) may be most effective for treating amyotrophic lateral sclerosis (ALS) in people with a shorter history of the disease, namely those diagnosed less than 18 months before receiving the treatment, a new analysis shows. The analysis was presented at the…
Researchers with Orion Corporation presented new data on levosimendan‘s mechanism of action, and potential biomarkers of amyotrophic lateral sclerosis (ALS) progression. The data were recently presented in four posters at the 30th International Symposium on ALS/MND, held Dec. 4–6, in Perth, Australia. Levosimendan, a small molecule that acts…
A new ALS Association partnership with the ALS Investment Fund II seeks to raise $100 million in new funding to advance amyotrophic lateral sclerosis (ALS) therapy development. This second venture fund follows one that originated in the Netherlands and raised $25 million in 2.5 years to support…
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…
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Reldesemtiv Effects Maintained When Used on Top of Rilutek or Radicava, New Trial Analysis Shows
Treatment with oral reldesemtiv appears to slow the deterioration of function — including breathing capacity and muscle strength — in amyotrophic lateral sclerosis (ALS) patients, regardless of whether or not they were taking additional medications such as Radicava (edaravone) or Rilutek (riluzole), a new…
With a $100,000 grant, the FTD Disorders Registry has received a boost from CurePSP, a nonprofit organization working to enhance awareness, education and care for prime-of-life neurodegenerative diseases. Launched in 2017 by the Association for Frontotemporal Degeneration (AFTD) and the Bluefield Project to Cure FTD — with…
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…
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…
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