Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes that cause at least half of the 7,000 rare diseases currently known — was the focus of a discussion by three top New York geneticists. The Feb. 28 conference,…
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IBIO-CFB03, an investigational fibrotic therapy, was selected as iBio’s lead candidate for further development, the company announced in a press release. The product may be a new treatment option for idiopathic pulmonary fibrosis (IPF) and scleroderma. Fibrosis is characterized by inflammation and buildup of collagen…
When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress — SMARD, for short. Hunter Pageau, a 12-year-old boy from North Haven, Connecticut, is one of only 80 people in the world known to have SMARD, a motor neuron disease…
HGE Health of Philadelphia is partnering with the Temple Lung Center to offer a telemedicine disease management program for idiopathic pulmonary fibrosis (IPF) patients in the United States and elsewhere. The healthcare technology company already has a similar program for patients with chronic obstructive pulmonary disease, or COPD. The Temple Lung Center, part…
Dentists and other dental healthcare personnel could be at risk of developing idiopathic pulmonary fibrosis at work, the U.S. Centers for Disease Control and Prevention (CDC) reports. An article about the danger appeared in CDC’s Morbidity and Mortality Weekly Report. It was titled “Dental Personnel Treated for Idiopathic…
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…
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…
