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…
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…
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