News

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

Electrocardiograms to Monitor Heart Health Advised for SSc-PH Patients

Changes from the norm in the electrical conduction of the heart — called cardiac axis deviations — are “common” and indicative of poorer outcomes in people with systemic sclerosis-related pulmonary hypertension (SSc-PH), a study reported. Because cardiac axis deviations are easily measured by electrocardiography (ECG), monitoring for such deviations…

Nominations Open for 2022 Eurordis Black Pearl Awards

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

Stem Cell-derived Tiny Vesicles Lessened Fibrosis, Inflammation

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…

Skin Fibrosis Worse When Lungs Involved, Study Finds

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…

Scleroderma Foundation More Than Doubles Research Funding to $2.7M

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…