News

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Hormones, Signaling Proteins in SSc Altered With BMI, Analysis Finds

Altered levels of immune signaling proteins (cytokines) and fat-based hormones in systemic sclerosis (SSc) are associated with changes in body mass, a blood analysis determined. Researchers say their findings suggest that “an abnormal twist … takes place in SSc” between the immune signaling proteins, these hormones, and body mass index or BMI,…

Pitt Study Testing COVID-19 Vaccines in Autoimmune Diseases

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…

Group Focuses on Rare Disease Clinical Trial Participation

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

Paquinimod, Possible Scleroderma Therapy, Shows Promise in Small Trial

Eight weeks of treatment with a small molecule called paquinimod significantly reduced the number of myofibroblasts — cells that drive scarring — in people with scleroderma, a small clinical trial reported. Findings also suggested that paquinimod may lessen type I interferon (IFN) activity, a relevant scleroderma biomarker, and appeared…

EnZen Invests in Thirona to Help Develop Topical Fibrosis Therapy

Thirona Bio has partnered with EnZen Therapeutics to accelerate the development of its experimental topical therapy FBM5712 for scleroderma and other conditions associated with scarring (fibrosis). EnZen’s investment in Thirona, part of a convertible promissory note arrangement, may be converted into equity in the long run. The companies…

Trial of COVID-19 Booster Vaccine Opens in Autoimmune Diseases

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…