Mitsubishi Tanabe Pharma America (MTPA) and Soleo Health have entered into an agreement that will facilitate Soleo sharing real-world data about the use of MTPA’s Radicava (edaravone) in amyotrophic lateral sclerosis (ALS) patients. Radicava was approved for the treatment of ALS by the…

If my husband, Todd, did not have ALS, there would still be someone else with ALS. And there’s cancer. Childhood illness. Tragic accidents. But winter has arrived on the Keweenaw Peninsula, and I’m compelled to get out and delight in God’s creation. Would He have made nature so beautiful…

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…

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…

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.

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…

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, “…