Insufficient intake of nutrients and energy (malnutrition) and weight loss are common in people with amyotrophic lateral sclerosis (ALS). Decreased food intake occurs mainly because of symptoms such as difficulty swallowing, as well as issues with hand grip and movement. In addition, digestive symptoms can…
Gleevec (imatinib mesylate), marketed by Novartis, is an oral medication approved by the U.S. Food and Drug Administration (FDA) for treating certain types of cancer, such as leukemia and gastrointestinal stromal tumors (GISTs). Gleevec also is being investigated in preclinical studies as a potential treatment for…
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease characterized by the death of motor neurons in the brain and spinal cord. ALS causes muscle stiffness, twitching, and weakness in its early stages. As the disease progresses, patients have difficulty speaking, swallowing, and breathing, in addition to body…
A 1992 episode of the hit television series “Seinfeld” is a “life-imitating-art” story titled “The Pitch.” The character of George Costanza (with brief help from Jerry Seinfeld) attempts to explain the concept for a new show to fictional NBC executive Russell Dalrymple: George: I think I can sum…
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…
“What one man can do, another can do.” So said actor Anthony Hopkins, playing the role of Charles Morse in the movie “The Edge.” He used that mantra as inspiration to fell a rogue, predatory grizzly bear. It may be hyperbolic to equate the difficulties of securing one’s…
Living with ALS certainly has its challenges, which lead many of us to rely on workarounds. These are creative, temporary solutions that solve an everyday problem; I first wrote about mine in “The ALS Workaround Dilemma.” But the key word here is “temporary,” because workarounds run the risk…
About a year ago, I wrote about how ALS patients may receive home healthcare on an uninterrupted basis, with expenses covered by Medicare. Since then, there has been both good news and bad news. The good news is that I recently “celebrated” my two-year anniversary of getting that coverage.
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…
