News

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…

The Phase 3 SENSCIS trial reached its primary endpoint by showing that Ofev (nintedanib) slows the decay in forced vital capacity (FVC) — a measure of lung function — in people with systemic sclerosis (SSc)-associated interstitial lung disease (SSc-ILD). The results of the trial were published in the article…

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 the…

Enrollment has been completed in an ongoing Phase 3 clinical trial assessing the safety and efficacy of experimental therapy lenabasum in patients with systemic sclerosis (SSc). Corbus Pharmaceuticals, lenabasum’s developer, expects to announce top-line results from the study during the summer of 2020, and hopes they will…

Combined pulmonary fibrosis and emphysema (CPFE) impairs lung function and decreases survival rates of systemic sclerosis (SSc) patients, a retrospective analysis shows. The study “Overall mortality in combined pulmonary brosis and emphysema related to systemic sclerosis” was published in the journal Rheumatic and Musculoskeletal Diseases. SSc is caused…

Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.

Corbus Pharmaceuticals is adjusting the primary efficacy outcome of the ongoing and global RESOLVE-1 Phase 3 trial of lenabasum as a possible treatment for diffuse cutaneous systemic sclerosis (SSc). The change affects only U.S. sites involved in the study and was recommended by regulatory officials. The adjustment, a change…

The U.S. Drug Enforcement Administration (DEA) has cleared the use of the active ingredient in EHP-101, an investigational therapy for treating multiple sclerosis (MS) and systemic sclerosis (SSc), according to Emerald Health Pharmaceuticals, the therapy’s developer. After a detailed review, the regulatory agency has determined that the active pharmaceutical…

Rituximab, an immunosuppressant antibody, alleviates skin fibrosis in patients with systemic sclerosis (SSc) and was also found to be generally safe, according to a study. The study, “Outcomes of patients with systemic sclerosis treated with rituximab in contemporary practice: a prospective cohort study,” was published in the…

Epigenetic changes — chemical modifications in DNA that affect gene expression (the process by which information within a gene gives rise to a functional product) — may be involved in the development of systemic scleroderma, a study says. The findings of the study, “Integrative analysis of DNA methylation…