In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…
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Increasing the production of lipids in the lungs may help slow pulmonary fibrosis progression and potentially lead to new treatment options, a study suggests. The study, “Lipid Synthesis is Required to Resolve ER Stress and Limit Fibrotic Responses in the Lung,” was published in the American…
The Pulmonary Fibrosis Foundation (PFF) has named Pauline Bianchi, a veteran nurse and pharmaceutical industry expert, its vice president of research and development. Two of her roles will be overseeing the PFF Care Center Network and PFF Therapeutics Network, key elements of the organization’s strategy to improve care and…
At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…
Using statins to prevent cardiovascular disease is safe for idiopathic pulmonary fibrosis patients, according to an analysis of trial data on the IPF therapy Ofev (nintedanib). Statin therapy does not contribute to lung function decline or diminish Ofev’s effectiveness in IPF patients, researchers said. Their analysis of data from…
The Feldman Family Foundation will hold its 5th Annual Texas Hold ’Em poker tournament to raise funds for pulmonary fibrosis research on Saturday, March 10, in the Chicago area. Foundation officials said the event will be in the grand ballroom of the Chevy Chase Country Club in Wheeling, Illinois. It…
On last week’s Rare Disease Day, held Feb. 28, Boehringer Ingelheim launched a new campaign titled “Why Wait in IPF?” to raise awareness about the importance of early diagnosis of idiopathic pulmonary fibrosis (IPF) and treatment with anti-fibrotic drugs. Even though international guidelines recommend treatment with anti-fibrotic drugs…
An engineered protein called AD-114, being tested to treat idiopathic pulmonary fibrosis, helped to prevent inflammatory cells from infiltrating and collagen from accumulating in the lungs in a mouse model of the disease. These findings were part of a collaborative preclinical study conducted by researchers at Alfred Hospital and Monash…
Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…
A new subtype of stem cells that have the unique ability to perform two distinct functions at the same time was recently discovered by researchers at University of Queensland (UQ) in Australia. These cells can generate both endothelial and mesenchymal cells, meaning they hold the potential to regenerate or…
