Genomic risk scores (GRS), which predict the likelihood of developing a condition based on a person’s genetic profile, successfully distinguished people with scleroderma from those with other immune-mediated inflammatory diseases or healthy individuals, a study…
Elevated blood levels of the S100A12 protein, a type of danger signal released by skin cells, may contribute to skin scarring and interstitial lung disease (ILD) in people with scleroderma, a study suggests. The study, “Serum S100A12 levels: Possible association with skin sclerosis and interstitial…
Treatment with abatacept led to a reduction in skin stiffening and disability, as well as better overall health, in people with early diffuse cutaneous scleroderma (dcSSc) who participated in the yearlong Phase 2 ASSET trial and its six-month extension, new 18-month data show.
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
Using a computer-based analysis to profile gene activity in blood samples may help identify the severe scleroderma patients who are most likely to respond to a stem cell transplant, a study suggests. That, in turn, will ultimately lead to better outcomes and faster improvement post-transplant for those who…
A Phase 1/2 clinical trial of FCX-013, Castle Creek Biosciences‘ experimental gene therapy for moderate to severe localized scleroderma, has dosed the first participant. The study (NCT03740724), which is expected to enroll up to 10 adults with localized scleroderma,…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
Autologous hematopoietic stem cell transplant (AHSCT) — replacing patients’ damaged bone marrow with their own healthy blood stem cells — improves health-related quality of life among people with scleroderma to a greater extent than conventional treatment, a study shows. The study, “Autologous hematopoietic stem cell…
Talaris Therapeutics has raised $115 million in financing, which will be used to support the clinical development of its lead therapeutic candidate, FCR001, designed to promote immune tolerance and allow patients to stop taking immunosuppressive medications after a transplant. The newly raised funds…
A Phase 3 trial testing iloprost, known as CIVI030, for the treatment of Raynaud’s phenomenon due to scleroderma has resumed patient enrollment, according to the therapy’s developer, CiVi Biopharma. The trial (NCT04040322), named AURORA and sponsored by Civi’s subsidiary Eicos Sciences, launched…
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