Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

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…

Mitsubishi Tanabe Pharma America (MTPA) has launched a new database that locates healthcare providers, area infusion treatment centers, and in-home infusion services, with the goal of providing a one-stop convenient resource for amyotrophic lateral sclerosis (ALS) patients and caregivers. The nationwide directory is available within the ALS…

I’ve always believed that I was open to new ways of doing things, but having ALS has put me to the test. My pride and a little denial have often gotten in the way of my seeing the obvious solutions to a problem. Thank goodness an “aha” moment came…

In observance of ALS Awareness Month, the ALS Therapy Development Institute (ALS TDI) has launched a campaign aimed at sparking more discussion about the progressive neurological disease. A leading non-profit drug discovery center focused solely on amyotrophic lateral sclerosis (ALS), the institute hopes to build on the awareness-promoting…

NPT520-34, an investigational small molecule for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS), is being tested on healthy individuals in a new Phase 1 clinical trial, its developer Neuropore Therapies has announced. Brain inflammation is a common feature among many neurodegenerative disorders, including Parkinson’s,…

New research details some of the clinical and genetic changes and imaging features seen in patients with amyotrophic lateral sclerosis type 4 (ALS4), a subtype of juvenile amyotrophic lateral sclerosis. This information could help clinicians quantify disease severity and track its progression in patients. The data were presented…

Only a minority of amyotrophic lateral sclerosis (ALS) patients are concerned about a possible negative impact of genetic testing on their well-being and that of their family, according to the results of a small survey in the Chicago area. The survey’s findings, presented in a poster session at the…

Reldesemtiv (formerly known as CK-2127107) reduces lung function decline and overall disease progression, without significant adverse events, in people with amyotrophic lateral sclerosis (ALS), a Phase 2 trial shows. The study, however, did not meet its primary endpoint. The new findings from the…

Making digital art can be beneficial for the psychological health of people with amyotrophic lateral sclerosis (ALS), a proof-of-concept study showed. The findings, “BRIDGE: Dynamic Imagery and Art Therapy in ALS- A Clinical Study to Improve Patient Expression and Wellness,” were presented in a poster session at…

A vitamin D activator molecule known as CYP27B1 is a biomarker of muscle alterations in patients with amytrophic lateral sclerosis (ALS) and may be a marker of clinical progression of the disease, according to new research. The findings, “The Vitamin D Activator, CYP27B1, is a Novel…