News

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…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

Following the onset of the COVID-19 pandemic, people with scleroderma experienced substantial increases in anxiety, but not depression, a new study has found. Results also show that older patients and those with greater financial resources experienced smaller increases in anxiety. The study, “Changes in mental health…

A new computer software, called Capillary.io, uses artificial intelligence (AI) to aid in diagnosing scleroderma at early disease stages. The tool, developed in partnership with the Sociedad Española de Medicina Interna (SEMI), was designed specifically to improve a non-invasive, highly sensitive and inexpensive technique — called nailfold capillaroscopy — used by…