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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…
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,…
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…
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…
Sanofi has entered into a merger agreement with Kadmon, which is developing belumosudil (KD025), an oral investigational treatment for people with the diffuse cutaneous form of systemic sclerosis (dcSSc). Belumosudil, sold under the brand name Rezurock, was recently approved for patients, 12 years and older, with chronic…
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…
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