Patricia Inácio, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Gesynta Pharma’s GS-248, its oral therapy for people with systemic sclerosis (scleroderma). Orphan drug status aims to encourage therapies for rare diseases through benefits such as seven years of market exclusivity and exemption from FDA fees. “The…

High levels of a form of the CD146 protein, normally found on endothelial cells — those that line blood vessel walls — may be a potential biomarker in identifying people with systemic sclerosis (SSc), a study suggests. Certain forms of the protein are implicated in lung scarring (pulmonary fibrosis)…

People with systemic sclerosis (SSc) vaccinated for COVID-19 fare well, with no serious adverse events and a low rate of reported flares, according to an international survey. “To our knowledge, this is the first large study that details experiences with COVID-19 vaccines in systemic sclerosis. Vaccination was safe in this group,”…

Poor oxygenation in skeletal muscles — those used in voluntary movements — contributes to impaired exercise capacity in people with scleroderma, a study suggests. The study, “Reduced exercise capacity in patients with systemic sclerosis is associated with lower peak tissue oxygen extraction: a cardiovascular magnetic resonance-augmented cardiopulmonary exercise…

Chemomab Therapeutics is teaming up with the University of Leeds to investigate the role of the protein CCL24 in scleroderma (SSc) — particularly how it contributes to blood vessel damage. Their findings may aid in the clinical development of CM-101, Chemomab’s first-in-class antibody designed to target and…

Having pulmonary hypertension shortens survival in people with scleroderma, also called systemic sclerosis (SSc), according to a Brazilian study that followed patients for up to three years and used a computer algorithm to analyze the results. Specifically, this lower survival rate in SSc patients with pulmonary hypertension was…

The fecal microbiome — the community of microbes present in stools — of scleroderma patients is more diverse than that of healthy people, according to a Canadian study. The findings also showed that the diversity and abundance of bacterial species in the stools was higher among patients with an abnormal…

Sexual dysfunction is common among both men and women with scleroderma (SSc), a review study says. According to the researchers, closer consideration of sexual dysfunction is warranted as it impairs patients’ quality of life. “Paying greater attention to the sexual function of patients with SSc is meaningful because sexual…

An international survey found that the ability to use one’s hands, physical symptoms, and the chance to prevent more severe problems are among the top factors taken into account by scleroderma patients when considering a therapeutic regimen for Raynaud’s phenomenon. Raynaud’s is a condition in which the fingers and toes…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…