Marisa Wexler, MS, senior science writer —

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…

Most scleroderma patients who responded to a U.S.-based online survey report being satisfied with their current medications and are not considering switching treatments. However, only about half of survey respondents reported being satisfied with their overall life quality, or optimistic about the future, perhaps highlighting gaps in care for…

A new study being conducted at the University of Pittsburgh is testing the safety and effectiveness of COVID-19 vaccines in people with scleroderma and other autoimmune diseases. The CoVER study seeks to enroll adults, 18 or older, who have rheumatoid arthritis, Sjogren’s syndrome, myositis, or…

An enrolling clinical trial is assessing whether a “booster shot” of a COVID-19 vaccine can improve the immune response in people with scleroderma and other autoimmune diseases who did not respond, at all or optimally, to their original COVID-19 vaccine regimen. The study also will test whether temporarily stopping…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Galapagos NV and Gilead Sciences are discontinuing all clinical trials with the investigational medication ziritaxestat. This includes the long-term extension of the Phase 2a NOVESA trial in scleroderma, as well as the Phase 3 ISABELA program in idiopathic pulmonary fibrosis (IPF). The decision is based on…

Small genetic variations that alter gene activity could play a role in the development of scleroderma and open new avenues for treating the disorder, a study indicates. Many of the genes identified through this work also show unusual levels of activity in tissues often affected in this disease. The study,…

Scleroderma treatment TMB-003, which aims to improve patients’ quality of life by reducing the scarring that characterizes the autoimmune disorder, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). This designation is awarded to investigational therapies intended to improve treatment for rare diseases,…

A new documentary, “Beyond Breathless,” will showcase the journey of those affected by interstitial lung disease (ILD) through the real experiences of people living with rare lung diseases, their loved ones, and healthcare professionals. The documentary, by pharmaceutical company Boehringer Ingelheim, will premier at noon EST Saturday (Dec. 5) on…