Under-the-skin treatment with tiny vesicles produced by mesenchymal stem cells significantly lessened skin abnormalities and scarring, while reducing markers of disease and inflammation, in a mouse model of scleroderma, a study showed. Notably, these beneficial effects — similar to those of treatment with the bone marrow-derived mesenchymal stem cells…
Scleroderma involving the lungs is associated with a greater degree of skin scarring, or fibrosis, according to a new research model that seeks to predict a person’s risk of organ-specific complications involved in that disorder. The study, “Correlation Between Skin and Affected Organs in 52 Sclerodermic Patients…
The Scleroderma Foundation is announcing that it has more than doubled its annual research funding, to $2.7 million, and increased the amount of its grant awards by $50,000, to $200,000. To accommodate the change in funding opportunities — effective for the 2022 grant-funding cycle — the nonprofit organization has…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
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.
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