News

As part of a new agreement with AdVita Lifescience, Relief Therapeutics will acquire the intellectual rights for the specifications and potential applications of an inhaled formulation of RLF-100 (aviptadil). Relief has been collaborating with NeuroRx to advance the development of RLF-100 to treat patients with severe…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

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)…