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Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

EHP-101, an oral cannabidiol treatment candidate for people with scleroderma (SSc) and multiple sclerosis (MS), was found to be safe and well-tolerated in a Phase 1 clinical trial in healthy volunteers. These results clear the way for Phase 2 trials, now in planning stages, to test the effectiveness…

The European Scleroderma Trials and Research Group (EUSTAR) revised activity index scale, known as EUSTAR-AI, is the best tool for predicting the short-term risk of disease progression and severe organ involvement in scleroderma (SSc) patients diagnosed within the last five years, a study reports. It had a better predictive…

Use of vasodilators — medications that trigger the widening and relaxing of blood vessels — may help to prevent heart problems in people with systemic scleroderma, a study suggests. Low-dose aspirin was also found to offer some protection against some forms of cardiac disease, its researchers said, recommending more…

Higher than usual serum levels of uric acid are linked to a greater risk of pulmonary arterial hypertension (PAH) among people with scleroderma, according to new research. Its findings also showed an association between higher levels of this blood biomarker and both disease severity and mortality in scleroderma-associated PAH…

Patient-reported symptoms — specifically those related to blood vessel, skin, and lung function — may be used to indicate changes in disease activity in people with systemic sclerosis (SSc), an Australian study suggests. The study, “Can patient-reported symptoms be used to measure disease activity in systemic…

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…

Evidence of pleuroparenchymal fibroelastosis (PPFE) — a rare type of interstitial lung disease — in people with systemic sclerosis (SSc) carries a negative prognosis, a study shows. Therefore, SSc patients with PPFE should be closely monitored and receive regular follow-up by their healthcare team. The study, titled “…

Loss of muscle mass and strength — known as sarcopenia — may affect up to 55% of patients with systemic sclerosis (SSc), a study suggests. Moreover, sarcopenia was found to be associated with several clinical and nutritional parameters of disease severity. The study, “Sarcopenia in systemic sclerosis: the impact…

Levels of a pro-inflammatory signaling molecule, called interleukin (IL)-6, are significantly high in skin lesions and blood samples of patients with systemic sclerosis (SSc), especially among those with diffuse cutaneous SSc, a small Japanese study reports. The study, “IL-16 expression is increased in the skin and sera of…