News

The American Lung Association and Foundation for Sarcoidosis Research are working together during April’s Sarcoidosis Awareness Month to offer U.S. sarcoidosis patients and caregivers additional support, making available in-person support groups and online tutorials by specialists. As part of the activities of Sarcoidosis Awareness Month, the associations welcome…

Patients with advanced sarcoidosis have limited daily life physical activity, mostly because their lungs’ maximal oxygen uptake is reduced, a study shows. The findings of the study, “Daily life physical activity in patients with chronic stage IV sarcoidosis: A multicenter cohort study,” were published in the journal Health…

Fatigue is the most commonly reported symptom by sarcoidosis patients in Denmark, Germany, and the Netherlands, according to a survey conducted by researchers from those countries. Nerve damage-related symptoms are a close second. The study, “The Burden of Sarcoidosis Symptoms from a Patient Perspective” was published in the journal…

A study reporting the case of a sarcoidosis patient infected with the bacteria Borrelia burgdorferi highlights the importance of maintaining an increased awareness about potential infectious conditions, such as Lyme disease, in patients with new onset of sarcoidosis. The case study, “Systemic Sarcoidosis Associated with Exposure to Borrelia burgdorferi in a…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bathdissertation, she analyzed Brexit’s long-term impact…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

The investigational therapy ATYR1923 for pulmonary sarcoidosis may help decrease lung inflammation by halting the infiltration of immune cells called neutrophils into the lungs, according to a preclinical study in mice. Suzanne Paz, PhD, from the therapy’s developer aTyr Pharma, presented the findings in a poster, titled “…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…