News

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

The Scleroderma Lung Study III (SLSIII), a Phase 2 clinical trial of oral CellCept (mycophenolate mofetil) alone or in combination with Esbriet (pirfenidone) in  scleroderma-associated interstitial lung disease (ILD) is enrolling patients. The trial (NCT03221257) is taking place across 17 clinical sites in the U.S., including the University…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

People with scleroderma who lack disease-related autoantibodies show a clinically distinct profile, which includes younger age at disease onset, and differences in skin thickness and the proportion of patients with diffuse disease, a Japanese study has found. The study, “Clinical features of Japanese systemic sclerosis…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

Pneumonia and sepsis are the most common serious infections among scleroderma patients needing hospitalization in the U.S., a study reports. Its findings also show that while the length of time these people remains in a hospital dropped slightly from 1998 to 2016, their hospital charges rose considerably. The study,…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

Long stretches of DNA repeats in the FLI1 gene — which gives instructions for a protein that controls the activity of genes — are linked to an increased risk for scleroderma and with greater skin thickness in people with the disorder, a study suggests. Titled “Association…

The first patients have been dosed in a Phase 2a clinical trial of EHP-101, Emerald Health Pharmaceuticals‘ investigational cannabidiol-derived treatment for diffuse cutaneous systemic sclerosis (dcSSc), the company announced. So far, the randomized, placebo-controlled trial (NCT04166552) has…

Talaris Therapeutics plans to initiate a Phase 1/2a trial to test its investigational cell therapy, FCR001, in people with diffuse cutaneous systemic sclerosis (dcSSc), the company has announced. The trial follows the recent approval of an investigational new drug application for FCR001 by the U.S.