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Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

Levels of a subtype of immune B-cells in the blood are increased in some people with scleroderma and associated with lower respiratory function, higher blood pressure in the lungs, and more kidney damage, a study reports. The study, “CD21-low B cells in systemic…

Using vitamin E, pentoxifylline, and the TheraBite Jaw Motion Rehabilitation System may help reduce trismus — commonly called lockjaw — in people with scleroderma, a study in two patients suggests. The research, “Dental management of scleroderma patients using pentoxifylline plus vitamin E with and…

Emerald Health Pharmaceuticals (EHP), the company developing the synthetic cannabis-derived EHP-101 as a treatment for scleroderma, has been granted six patents related to 25 new molecules derived from cannabidiol (CBD) and cannabigerol (CBG). The new patents were granted in Japan, Israel, Australia, and Russia. Both CBD…

Higher-than-normal blood levels of squamous cell carcinoma antigen 1 (SCCA1), a pro-fibrotic protein, are associated with a greater risk of interstitial lung disease (ILD) among people with systemic sclerosis (SSc-ILD), a study suggests. This finding points to SCCA1 blood levels as a potential biomarker for early detection of SSc-ILD,…

Heart and kidney imaging may help detect fibrosis, or scarring, in asymptomatic people with scleroderma, and could be useful in routine evaluations for people with the disease, a study suggests. The study, “Late Gadolinium Enhancement in Cardiac Magnetic Resonance Imaging Is Associated with High Renal Resistive Index…

A new non-invasive ultrasound imaging technique can detect changes in skin elasticity that are predictive of disease progression in scleroderma, a recent pilot study suggests. Early detection of these changes can make a difference in the diagnosis and treatment of the chronic connective tissue disease,…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

One year of treatment with Tracleer (bosentan) significantly reduces the severity of patient-reported skin symptoms in people with scleroderma, a new study suggests. Yet, these benefits are diminished among those with more severe complications before treatment. The study, “A patient-centered approach to the burden…

EHP-101, an oral therapy based on cannabidiol (CBD), has been placed on fast track development by the U.S. Food and Drug Administration (FDA) as a potential treatment of systemic…