News

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…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

The American Thoracic Society (ATS) Foundation Research Program and the Foundation for Sarcoidosis Research (FSR) have awarded a Foundation Partner Grant to Nicholas Arger, MD, at the University of California San Francisco, to study the role of a specific population of immune cells in sarcoidosis-related…

aTyr Pharma announced a collaboration and license agreement with Kyorin Pharmaceutical to develop and commercialize ATYR1923, its potential disease-modifying treatment for those with interstitial lung diseases (ILDs), including pulmonary sarcoidosis, in Japan. ATYR1923 is a fusion protein that contains a portion of a human antibody linked to…

The transplantation program at Stanford Medicine has hit a milestone with its 1,000th procedure — performed on a 54-year-old woman with sarcoidosis. The surgery, carried under the Heart-Lung & Lung Transplantation Program at Stanford Health Care, is now part of the 750 lung transplants and 250 heart-lung…