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…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
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…
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…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
Supervised rehabilitation combining a whirlpool bath with active exercises and hand and elbow massages may improve hand and overall function in people with scleroderma, a study from Poland reported. Its findings suggest that a four-week rehabilitation program may be enough for these benefits to last up to six months…
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…
Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…
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…
High levels of a protein known as CTRP9 appear to indicate worsening lung function in patients with systemic scleroderma (SSc), according to a recent study. The discovery suggests that CTRP9 may be a useful biomarker for the loss of lung function in SSc. Such biomarkers are sorely needed, researchers…
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