News

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

Boehringer Ingelheim‘s Ofev (nintedanib) has been approved in the U.S. as the first treatment for chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype. This approval by the United States Food and Drug Administration (FDA) includes sarcoidosis, autoimmune ILDs, unclassifiable ILDs, chronic hypersensitivity pneumonitis,…

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…

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…

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…

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…

Researchers have discovered that patients with pulmonary sarcoidosis have an imbalance in two subsets of immune cells, namely B-cells and follicular T-helper cells — findings that support the potential role of autoimmune mechanisms in sarcoidosis development. The study, “Imbalance in B cell and T Follicular Helper…

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…

The arthritis medicine Xeljanz (tofacitinib) may be an effective therapeutic alternative to conventional medications for the treatment of multiorgan sarcoidosis. This finding was described in the study, “Treatment of Multiorgan Sarcoidosis With Tofacitinib,” published in the journal ACR Open Rheumatology. Sarcoidosis, an inflammatory condition…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…