News

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

A case study diagnosing pulmonary sarcoidosis in a patient complaining of chest pain and shortness of breath highlights the importance of including sarcoidosis in the differential diagnosis of chest pain. The study, “Sarcoidosis Presenting as Acute Respiratory Distress Syndrome,” was published in the journal Clinical Practice and…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

Researchers in Japan reported a case of sarcoidosis associated with a specific bacterium and complicated by an acute bird-related — pigeons, in this case — inflammatory lung disease called hypersensitivity pneumonitis. A distinct type of granuloma specifically linked to the man’s bird-related disease was also, possibly for the…

Although uncommon, some people with cancer will develop granulomas, the clumps of immune cells that are a hallmark of sarcoidosis — and those who do are more likely to have better survival outcomes, a new study reports. The study, “Presence of concurrent sarcoid-like granulomas indicates better…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to XTMAB-16, Xentria’s investigational TNF-alpha inhibitor for the treatment of sarcoidosis. The designation is given to candidate therapies with the potential to be safe…

Three months of treatment with the common, generic antibiotic azithromycin safely reduced the number of coughs and cough severity, and improved the quality of life in adults with pulmonary sarcoidosis, a small, exploratory study concluded.  The findings…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…