A less toxic precursor formulation of the chemotherapeutic agent paclitaxel can reduce lung fibrosis in mice with pulmonary fibrosis, a study shows. The finding suggests that this compound, named baccatin III or BAC, has the potential to represent a new treatment strategy for…
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A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
Genetic variants of the MUC5AC gene may contribute to the development of idiopathic pulmonary fibrosis (IPF), a study suggests. The study, “Novel idiopathic pulmonary fibrosis susceptibility variants revealed by deep sequencing,” was published in the journal ERJ Open Research. The existence of a genetic cause underlying…
Distal airway stem cells (DASCs), which can differentiate into bronchioles and alveoli, were found to prevent fibrosis and support tissue regeneration in the lungs of mice. These findings were reported in the study “Distal airway stem cells ameliorate bleomycin-induced pulmonary fibrosis…
A treatment algorithm based on high-flow nasal cannula oxygen therapy reduces short-term mortality of people with idiopathic pulmonary fibrosis who develop acute respiratory failure, a study says. The findings of the study, “High-flow nasal cannula oxygen therapy to treat acute respiratory failure in patients with acute exacerbation…
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
Treatment with Esbriet (pirfenidone) slows the worsening of breathlessness in patients with more severe idiopathic pulmonary fibrosis (IPF), a post-hoc analysis of the ASCEND and CAPACITY trials shows. The study, “Effect of pirfenidone on breathlessness in patients with idiopathic pulmonary fibrosis,” was published in the European…
Note: This is the second article in a three-part series written in collaboration with respiratory therapist Mark W. Mangus Sr., RRT, RPFT, FAARC, and oxygen expert Ryan Diesem. The first part can be found here. When it comes to home improvement, people typically want three things from a contractor:…
The presence of auto-antibodies (self-directed antibodies) known as antineutrophil cytoplasmic antibodies do not correlate with disease severity or survival in North American patients with idiopathic pulmonary fibrosis (IPF), according to a new study. The study, “Prevalence and Clinical Significance of Antineutrophil Cytoplasmic Antibodies in North American Patients with…
People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
