Despite immunosuppressive therapy, skin and lung involvement still progresses in about 20% of people with early or at-risk diffuse cutaneous systemic sclerosis (dcSSc) within the first three years, according to a study in the U.S. In addition, most people in the at-risk group went on to develop dcSSc during…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
An international survey found that the ability to use one’s hands, physical symptoms, and the chance to prevent more severe problems are among the top factors taken into account by scleroderma patients when considering a therapeutic regimen for Raynaud’s phenomenon. Raynaud’s is a condition in which the fingers and toes…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
Blood levels of the protein endocan may be a potential biomarker in identifying people with scleroderma who are at risk for pulmonary arterial hypertension (PAH), a study reported. The study, which supported previous findings regarding this protein, was titled “Endocan and Circulating Progenitor Cells in Women with Systemic…
Gesynta Pharma has submitted an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) seeking approval to test its oral therapy, GS-248, in clinical trials with systemic sclerosis (scleroderma) patients. GS-248 is an experimental therapy being developed for conditions characterized by microvascular disease, including systemic sclerosis.
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
The amount of free light chain (FLC) molecules in blood and urine correspond to the severity of scleroderma, adding to the evidence supporting these molecules as biomarkers for early diagnosis and disease activity. The pilot study detailing this finding, “Serum and urine free light chains…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…
From joining a walk-a-thon to sharing videos of themselves saying the rare disease’s hard-to-pronounce name, supporters are marking Scleroderma Awareness Month, observed each June. World Scleroderma Day is June 29. The mission is to raise awareness among the general public and to educate policymakers, public authorities, industry representatives, scientists, and…
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