News

In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…

Hospitalization due to sarcoidosis has increased in the U.S., but in-hospital mortality has declined, according to a study from the University of Alabama at Birmingham (UAB). The data also showed that more than 30 percent of the hospitalized sarcoidosis patients had one or more cardiovascular complications. The research, “…

Experts recently reviewed the latest advances in pulmonary sarcoidosis research, highlighting possible causes of the disease, as well as potential treatment candidates. The article, “The Pathogenesis of Pulmonary Sarcoidosis and Implications for Treatment,” was published in CHEST Journal. Sarcoidosis is common in the lungs, characterized…

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

A small study of a group of patients in Canada who were showing the first signs of sarcoidosis suggests that isolated cardiac sarcoidosis, which refers to cardiac involvement without the disease clinically apparent elsewhere, may be rarer than previously reported. The study, “How common is isolated cardiac sarcoidosis? Extra-cardiac and…

In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…

A recent study has reported on a multiple sclerosis (MS) patient who developed pulmonary sarcoidosis after receiving the relapsing-remitting MS (RRMS) therapy daclizumab. The report “Pulmonary Sarcoidosis in a patient with Multiple Sclerosis on daclizumab monotherapy,” was published in Multiple Sclerosis and Related Disorders. Daclizumab is a…

Blood levels of a soluble protein can be used as a diagnostic test to confirm sarcoidosis in cases of acute uveitis, a test that is superior to the standard biomarker used, called angiotensin-converting enzyme (ACE). The results were reported in “Diagnostic…