News

A first patient has been enrolled in a pivotal Phase 2b clinical trial testing the safety and effectiveness of HZN-825, Horizon Therapeutics‘ oral investigational medicine for people with diffuse cutaneous systemic sclerosis (dcSSc), a form of systemic scleroderma. The trial (NCT04781543) aims to enroll 300 people, between…

Children with an extremely rare form of systemic scleroderma tend to experience longer diagnostic delays, are more likely to have heart involvement, and have worse clinical outcomes, according to a study of five children with the condition, which has no skin involvement. The study, “Systemic sclerosis…

Platelet inhibitors, or anti-platelet medication, is associated with a lower occurrence of digital ulcers — open sores on the fingers or toes — in people with systemic sclerosis (SSc), a study shows. These findings may “assist rheumatologists in decision making regarding the use of platelet inhibitors in SSc patients…

Black patients with scleroderma are at higher risk of mortality and serious complications such as interstitial lung disease than those of other racial or ethnic groups, a single-center U.S. study found. These findings may foster early monitoring of these patients as well as greater awareness among clinicians, researchers…

Over 230 national organizations signed a letter urging all 50 U.S. state governors to “maintain and expand” flexibility with licensure requirements for the duration of the COVID-19 pandemic to ease access to care. During the pandemic, governors used emergency authority to waive certain state licensure requirements, giving healthcare providers…

Infection was the leading cause of acute hospitalization and mortality in people with systemic sclerosis (SSc), a 10-year hospital study in Portugal found. Although most infection-related hospital admissions occurred in those with underlying lung disease, the scientists noted that a decrease in the total number of hospitalizations during the…

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…

Ofev (nintedanib) effectively slowed lung function decline in adults with interstitial lung disease (ILD) associated with systemic sclerosis (SSc-ILD) across patient groups based on autoantibody status and skin involvement severity, according to an analysis of the SENSCIS trial. These findings add to the trial’s top-line results,…

Cudetaxestat, a potential anti-scarring medication, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) for the treatment of scleroderma. “There are limited options and high medical need for patients suffering from systemic sclerosis [SSc, or scleroderma],” Daven Mody, vice president of regulatory affairs…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…