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, “…
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…
After my husband Todd’s ALS diagnosis, he said, “We need to sell the house and move near your parents.” He anticipated that their day-to-day support in rural northern Michigan would be more important than being near an ALS clinic in Milwaukee, Wisconsin. We appreciated the convenience of seeing Todd’s…
When I was diagnosed with ALS in 2010, my doctor told me there was one medication available to help slow the progression of the symptoms. But there was no cure. I left her office with a prescription for Rilutek (riluzole), the first treatment…
Small molecule inhibitors that block the activity of SARM1 — an enzyme that plays a key role in nerve cell degeneration — prevent the degeneration of axons in mice and cellular models of disease, a preclinical study has found. These findings may be the first steps towards the development…
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
I’m always pleased to read news of advancements in health and wellness for those of us living with ALS. But one statement always makes me laugh out loud: ALS patients shouldn’t exercise because they need to save their energy. More than 10 years of published research on exercise and…
