Having scleroderma increases the risk of developing diseases affecting blood vessels of the brain or heart, known as cerebrovascular and cardiovascular diseases, a review study found. Relative to controls, the risk of stroke — caused by poor blood flow to the brain — in scleroderma patients was 64% higher, whereas that of cardiovascular disease was 112% higher. “Our findings support increased clinical surveillance and consideration of preventative strategies in this high-risk population,” researchers in Taiwan wrote. Their study, “Association between systemic sclerosis and risk of cerebrovasc ..read more

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to BLR-200, a medication that BLR Bio — a company stemming from the Helix 51 biomedical incubator at Rosalind Franklin University — is developing for scleroderma. According to Ronald Kaplan, PhD, the university’s executive vice president for research, “BLR-200 has the potential to establish first-in-class clinical benefits by addressing multiple elements driving scleroderma and treating multiple organs negatively impacted by the disease.” Orphan drug status is a designation given by the FDA to investigational therapies ..read more

Levels of two blood proteins, calcitonin and SOST, may be markers of lung disease in people with scleroderma, a new study shows. “This study indicates that serum calcitonin and SOST levels are promising biomarkers for [scleroderma]-related PAH [pulmonary arterial hypertension] and ILD [interstitial lung disease], respectively,” the researchers wrote, though they noted that “further research is needed to verify these results and understand the underlying mechanisms.” The study, “Protein profiling in systemic sclerosis patients with different pulmonary complications using proteomic antibod ..read more

People with scleroderma who feel more socially isolated are more likely to report being dissatisfied with life, but this association is partially attenuated in those who show more resilience, according to a recent study. Resilience refers to the ability to adapt to and recover from challenging life experiences, such as living with a chronic disease. These findings suggest that more adaptive coping may help protect against the negative effects of perceived social isolation, and “support the promotion of social connection and resilience to enhance life satisfaction in people with [sclerode ..read more

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Cabaletta Bio’s cell therapy CABA-201 as a treatment for adults with hard-to-treat systemic sclerosis (SSc), or scleroderma. Orphan drug designation is intended to support the accelerated development of investigational treatments for rare diseases, defined as those affecting fewer than 200,000 people in the U.S. The designation provides Cabaletta with regulatory support, financial incentives, and seven years of market exclusivity if CABA-201 is approved. “Orphan drug designation is an important recognition for ..read more

Lung disease severity in scleroderma (SSc) patients may be linked to levels of immune cells known as follicular T-helper cells (Tfh cells), according to a small study. The findings “may guide the development of targeted therapies for this aspect of the disease,” the researchers wrote. Titled “The relationship between peripheral T follicular helper cells and disease severity in systemic sclerosis,” the study was published in the journal Clinical and Experimental Medicine. In scleroderma, the immune system produces self-targeting antibodies, or autoantibodies, that lead to hardening and fibrosi ..read more

Inflammatory arthritis — a condition marked by joint inflammation that causes swelling, pain, and damage — affects about one-third of people with scleroderma, also known as systemic sclerosis (SSc), and is associated with worse health-related quality of life, or HRQoL for short, according to a new study from Australia. “Our results reveal the significant impact that [inflammatory arthritis] has on patients, with the presence of [such inflammation] being negatively associated with employment, physical function, and HRQoL,” the researchers wrote. Given that such arthritis is treatable, “further ..read more

Cognitive difficulties affected half of all scleroderma patients in a recent study in Italy, while malnutrition was found in more than one quarter. While no correlation was seen between cognitive impairment and malnutrition in the 100 patients involved, the presence of these complications associated with greater functional disability and a poorer quality of life. Identifying them early is “pivotal to better address the chronic needs of patients affected by this disease,” researchers wrote in the study, “Role of cognitive impairment and malnutrition as determinants of quality of life in patien ..read more

Muscle disease is common in people with scleroderma and is associated with inflammation and specific end-organ involvement, according to an Australian study. The findings underscore the “clinical, functional, and prognostic importance of simple biomarkers ” for identifying SSc-related muscle disease, the study’s researchers wrote in “Proximal weakness and creatine kinase elevation in systemic sclerosis: clinical correlates, prognosis and functional implications,” which was published in Seminars in Arthritis and Rheumatism. Scleroderma, also called systemic sclerosis (SSc), is a chronic d ..read more

Certa Therapeutics’ FT011, an investigational oral therapy for systemic sclerosis (SSc), has been granted fast track designation by the U.S. Food and Drug Administration (FDA). It comes following Phase 2 clinical trial data that showed that FT011 was safe and well tolerated, and led to clinically meaningful improvements in lung function for some participants. The therapy also helped to ease physical disability for more than half of the patients in the small study. The FDA fast track designation, which provides incentives such as more frequent meetings with the regulatory agency and poten ..read more