News

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

New diagnostic guidelines for cardiac sarcoidosis yield a higher number of diagnoses and identify more cases of isolated cardiac sarcoidosis (iCS), according to a study in Japan. In cardiac sarcoidosis, inflammatory clusters of white blood cells — called granulomas — affect the heart but can also affect other…

Protalix BioTherapeutics and SarcoMed USA have entered into a non-binding agreement related to the development and commercialization of PRX-110 (alidornase alfa) for pulmonary sarcoidosis and related diseases, according to a Protalix press release. PRX-110 is currently being developed by Protalix as a potential therapy for…

More than 20% of sarcoidosis patients show signs of an immune system response against aluminum, beryllium, zirconium, and silica, supporting the idea that these compounds may be involved in the development of the disease in a subgroup of patients, according to findings from a research study. The study, “…

Women with sarcoidosis are more likely to die in hospital from heart complications and also to experience sudden cardiac death than male patients, while men are more likely to have a heart defibrillator device, a study reports. These findings highlight the importance of recognizing how sex differences…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

People with sarcoidosis-associated pulmonary hypertension who have reduced physical fitness or worse gas exchange capacity live significantly shorter times without requiring a transplant, a new study indicates. The study, “Physiological Predictors of Survival in Patients with Sarcoidosis Associated Pulmonary Hypertension,” was published in the European Respiratory…

People living with or affected by sarcoidosis are invited to apply for membership in the new patient advisory committee being established by the Foundation for Sarcoidosis Research, known as the FSR. The committee is being created to help establish an advisory board to further bring patients’ voices…

Severe fatigue affects more than 60% of pulmonary sarcoidosis patients and is associated with worse shortness of breath, daytime sleepiness, anxiety and depression, catastrophizing, functional impairments, and quality of life, according to a Dutch study. The research also showed similar findings for people with idiopathic pulmonary fibrosis (IPF),…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…