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…
Gene Therapy Takes Center Stage at 2019 NORD Summit
News
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…
As I approached the one-year anniversary of my husband, Todd’s, ALS diagnosis, I wrote in my journal: “I want to be happy, healed, and whole again. But the grief that I am experiencing is not something one can easily get over.” I read books such as “Getting…
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…
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…
It’s Thanksgiving Day tomorrow in the United States. Unsurprisingly, the evolution of its celebration has been shaped by our nation’s cultural dynamism. Rooted in religious ritual imported by the initial European settlers, it was institutionalized by the constitutional congress, with specific…
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…
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