News

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…

A form of ultrasound imaging called shear-wave elastography (SWE) is more sensitive for assessing changes in skin stiffness over time in scleroderma (SSc) patients than the current gold standard measure, a study suggested. Because a lessening in skin stiffness was also observed in healthy people, skin changes…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

Social distancing might thin the crowds, but it won’t stop the walks, runs, and other activities planned throughout June, in support of Scleroderma Awareness Month. The events, coordinated annually by the Scleroderma Foundation, aim to raise funds and awareness, as well as to encourage participation in patient advocacy. The…

The U.S. Food and Drug Administration has granted orphan drug designation to Theraly Fibrosis’ TLY012 for the treatment of systemic scleroderma. This designation is given to medications with the potential to treat rare conditions — those that affect fewer than 200,000 people in the U.S. Benefits include…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…