aTyr Pharma has dosed the first person in its Phase 1 study of iMod.Fc (ATYR1923) — an investigational therapy developed for patients with interstitial lung diseases, including sarcoidosis. The study is the first to test the compound in humans and will compare the treatment to a placebo in healthy…
News
The Oklahoma Medical Research Foundation (OMRF) is seeking volunteers to participate in sample donations for the launch of a new Sarcoidosis Research Clinic. Volunteers needed include healthy individuals and people with a sarcoidosis diagnosis. Both African-American and European-American individuals are eligible to participate. The clinic is the first…
The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…
The bacterium Propionibacterium acnes may cause ocular sarcoidosis, as researchers have detected high levels of the pathogen in retinal granulomas of patients with the condition. These findings suggest that new therapies for the disease may include antibiotics targeting the infection. The study, “Immunohistochemical Detection of Propionibacterium acnes in the…
Researchers from Turkey’s Ege and Istinye Universities reported the coexistence of sarcoidosis and gouty arthritis in a 45-year-old man. Their study, “Coexistence of Sarcoidosis and Gouty Arthritis.” appeared in the Spanish journal Reumatología Clínica. Sarcoidosis is a rare inflammatory disease characterized by the formation of granulomas in organs of…
Therapies used to treat pulmonary hypertension (PH), such as Revatio (sildenafil), provide only temporary benefit for patients with sarcoidosis-associated PH, according to a case report published in the journal Advances in Respiratory Medicine. The report demonstrates that such therapies fail to prevent disease progression and improve patients’ outcomes, highlighting the…
Treatment with medications for pulmonary arterial hypertension (PAH) do not significantly help people with sarcoidosis-associated pulmonary hypertension (PH), researchers from France’s University of Paris-Sud reported. Their study, “Management and long-term outcomes of sarcoidosis-associated pulmonary hypertension,” — published in the European Respiratory Journal — showed that while PAH-drugs improved some disease…
Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
A recent report in the journal Internal Medicine, published by the Japanese Society of Internal Medicine, described the unusual case of a 78-year-old woman who had a rare combination of pancreatic mass and nodules in the airways and lungs — all consistent with a diagnosis of sarcoidosis. Sarcoidosis is characterized…
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