Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

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…

Injection of human neural stem cells into the spinal cord of people with amyotrophic lateral sclerosis (ALS) was found safe and did not cause adverse effects even two years after the transplant, results from a Phase 1 clinical trial show. Trial findings were published in the study, “Results…

“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…

Although people who develop amyotrophic lateral sclerosis (ALS) have altered levels of several blood metabolites — products of our cells’ metabolism — before disease onset, these changes do not enable reliable identification of who is at risk of having ALS, according to a large study. The research, “…

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…

People with familial amyotrophic lateral sclerosis (ALS) related to the gene VAPB — known as ALS type 8 — can show subtle cognitive deficits and obvious behavioral changes, which may lead to clinically significant depression and anxiety, a Brazilian study shows. This data supports prior studies…

Using antibiotics may increase the risk of developing amyotrophic lateral sclerosis (ALS), with a higher number of prescriptions linked to greater risk, a nationwide study in Sweden suggests.  However, the researchers caution that more studies are needed to prove a direct,…

People with amyotrophic lateral sclerosis (ALS) who tolerated higher doses of tirasemtiv showed a trend toward slower decline in breathing muscle activity, although the difference was not statistically significant between those treated with the investigational therapy and those on a placebo, a Phase 3 clinical trial shows. These results may have…

Oxford BioDynamics has joined the REFINE-ALS study, a project designed to detect and measure the levels of specific biomarkers among people with amyotrophic lateral sclerosis (ALS), Mitsubishi Tanabe Pharma America (MTPA) announced. REFINE-ALS is sponsored by the MTPA and led by the Massachusetts General Hospital (MGH)…

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.