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Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…

Abatacept, a potential treatment for people with early-stage diffuse cutaneous systemic sclerosis (dcSSc), showed clinically significant improvements in patients’ functional ability and overall health, despite failing to significantly reduce skin thickening, a Phase 2 study concluded.  The study, “Abatacept…

The Arthritis Advisory Committee of the U.S. Food and Drug Administration (FDA) has recommended — via a 10-7 vote — the approval of Ofev (nintedanib) for the treatment of systemic sclerosis-associated interstitial lung disease (SSc-ILD). Ofev, marketed by Boehringer Ingelheim, is…

A subgroup of patients with rapidly progressive diffuse cutaneous scleroderma (dcSSc) experienced a relapse of skin manifestations shortly after stopping or lowering the dose of CellCept (mycophenolate mofetil), according to a five-year study. The findings also showed rapid improvements after restarting CellCept, and revealed that patients with faster…

Blood levels of three types of immune cells — neutrophils, lymphocytes, and monocytes — may be associated with digital ulcers and disease severity in systemic sclerosis, researchers report. Their study, “Association of simple hematological parameters with disease manifestations, activity, and severity in patients with systemic sclerosis,”…

Lipotransfer — a procedure in which fat tissue is injected into facial scar tissue — improved mouth function, appearance, and quality of life for people with scleroderma, a study says. The study, “Stem cell enriched lipotransfer reverses the effects of fibrosis in systemic sclerosis,” was published…

A new computerized method to analyze high resolution computed tomography (HRCT) scans may help in identifying interstitial lung disease at early stages in scleroderma patients, the research team that developed it reports. The tool was described in the study “Performance of a new quantitative computed tomography index for…

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…

Rituximab, an immunosuppressant antibody designed to target immune B cells, has widespread effects and decreases subtypes of other immune cells called T cells in the skin and blood of people with systemic sclerosis (SSc), new research shows. The study, titled “B cell depletion treatment decreases CD4+IL4+ and CD4+CD40L+…