News

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…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

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…