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…
Linaclotide, an oral medication to treat constipation, is safe and well-tolerated, and can be used to manage difficult-to-treat gastrointestinal (GI) complications in people with scleroderma, a study has found. Results show that low-dose treatment with linaclotide is effective and may be better tolerated, though some patients may require higher…
Mary J. Wheatley will succeed Robert J. Riggs as CEO of the Scleroderma Foundation, an organization dedicated to supporting scleroderma patients, promoting public education concerning the disorder, and helping to fund research into effective treatments and a possible cure. The change will be effective July 1. “This is…
People with weakened immune systems, such as those with scleroderma, should continue taking all COVID-19 precautions, even if fully vaccinated, according to guidelines from the Centers for Disease Control and Prevention (CDC). “The Scleroderma Foundation’s MSAB [Medical & Scientific Advisory Board] Leadership Committee agrees with the CDC…
A remote, peer-facilitated group intervention created to provide mental health and social support during the COVID-19 pandemic significantly eased anxiety and depression in adults with systemic scleroderma and mild anxiety, according to data from the SPIN-CHAT trial. Differences between the group of patients who underwent the one-month intervention…
Ofev (nintedanib) safely and effectively slowed lung function decline in people with interstitial lung disease (ILD) associated with systemic sclerosis (SSc-ILD), according to an interim analysis of the ongoing and long-term SENSCIS-ON study. “The SENSCIS-ON trial provides important new evidence about the longer-term use of nintedanib [Ofev] in people living…
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