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.
Obstructive sleep apnea (OSA) is associated with an increased risk of pulmonary hypertension in people with scleroderma, a study suggests. The study, “Impact of concomitant obstructive sleep apnea on pulmonary involvement and main pulmonary artery diameter in adults with scleroderma,” was published in the journal Sleep…
The American College of Rheumatology Combined Response Index in diffuse cutaneous systemic sclerosis (ACR CRISS) appears to be a consistent and reliable measure of how these patients feel and function, according to data recently presented by Corbus Pharmaceuticals. Notably, the research suggests that this composite measure, which is frequently used…
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…
Orencia (abatacept) — an approved medication for juvenile idiopathic arthritis and other inflammatory conditions — is a safe and effective treatment for localized scleroderma in both children and adults, according to a case series and review study. “This is the first published paper on abatacept as a…
CellCept (mycophenolate mofetil) and cyclophosphamide can both improve the health-related quality of life of people with systemic sclerosis-related interstitial lung disease, an analysis from a Phase 2 clinical trial shows. Findings were reported in the study, “Treatment With Mycophenolate and Cyclophosphamide Leads to…
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…
Francesco Boin, a nationally recognized rheumatologist, has taken over as director of the division of Rheumatology and of the new Scleroderma Program at Cedars-Sinai‘s Kao Autoimmunity Institute. Boin previously established the Scleroderma Center at the University of California, San Francisco, and was director…
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