The Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Ofev (nintedanib) to preserve lung function in people with systemic sclerosis (SSc)-associated interstitial lung disease (SSc-ILD) in the European Union. Boehringer Ingelheim, the company marketing Ofev, submitted the request for this indication in…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
A panel of experts in Europe defined a set of recommendations for the diagnosis and management of interstitial lung disease (ILD) in people with systemic sclerosis (SSc), based on a systematic literature review and expert consensus. The guidelines were outlined in a report, “The identification and management…
Inhibiting a protein known as KLHL42 may ease lung fibrosis (scarring) associated with systemic scleroderma (SSc), according to a recent study. The findings were published in an article, “Kelch-like protein 42 is a pro-fibrotic ubiquitin E3 ligase involved in in systemic sclerosis,” in the Journal…
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar. William Whitman…
A statistical model applied to a widely used measure of skin thickening in people newly diagnosed with systemic sclerosis (SSc) identified five classes that may predict organ involvement and survival rates. Using these predictive classes — based on the…
The types of autoantibodies a person has are a better predictor of scleroderma prognosis than whether the person has other connective tissue diseases, a study reports. The study, “Clinical characteristics and survival in systemic sclerosis-mixed connective tissue disease and systemic sclerosis-overlap syndrome,” was published in the journal…
A pre-clinical stage preceding the development of digital ulcers could provide a window of opportunity for treatment before serious tissue damage occurs in people with scleroderma, according to a new study. The study was summarized in a letter to the editor titled, “Patient experiences of digital ulcer development…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
A long RNA molecule called HOTAIR is produced in excess in people with scleroderma and controls the activation of myofibroblasts, key cells in the progression of this disease, according to a study. The finding expands the understanding of how to target biological drivers of scleroderma, and offers a potential…
Get regular updates to your inbox.