A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
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A new way of assessing ultrasound, a non-invasive technique using sound waves to produce pictures inside of the body, may be effective in diagnosing idiopathic pulmonary fibrosis (IPF), a study reported. This approach was able to quantify lung scarring, a hallmark of IPF, in a study in rats, as well…
All patients in a Phase 1/2 trial examining two doses of AP01, an inhaled version of pirfenidone for the treatment of idiopathic pulmonary fibrosis (IPF), are switching to the higher dose based on the recommendation of a data safety monitoring board (DSMB). Avalyn Pharma, AP01’s developer, reported that…
A Phase 2 study of NP-120 (ifenprodil) for chronic cough associated with idiopathic pulmonary fibrosis (IPF) has reached 25% of its enrollment target, Algernon Pharmaceuticals, the therapy’s developer, has announced. “I am very pleased to report that we have reached 25% enrollment in the study,” Christopher J. Moreau, CEO…
A protein-coding gene called Spred2 may contribute to pulmonary fibrosis (PF) development, potentially representing a new therapeutic target, according to the results of a new study. Titled “Spred2-deficiency enhances the proliferation of lung epithelial cells and alleviates pulmonary fibrosis induced by bleomycin,” the study was…
Treatment with inhalable bone morphogenic protein 4 (BMP4) — a soluble signaling molecule involved in the regulation of cellular identity — restored lung regeneration and improved lung function in a mouse model of idiopathic pulmonary fibrosis (IPF). The data showed that scar-forming cells in IPF lungs suppress BMP signaling…
People with idiopathic pulmonary fibrosis (IPF) who have three or more abnormally large lymph nodes in the area of the lungs and heart — a condition known as mediastinal lymph node enlargement (LNE) — have an increased risk of mortality and faster disease progression, a study found. The…
Esbriet (pirfenidone), an approved treatment for idiopathic pulmonary fibrosis (IPF), seems to be safe in IPF patients who also have non‐small cell lung cancer (NSCLC) when combined with cancer therapy. The finding is detailed in the study, “Safety and effectiveness of pirfenidone combined with carboplatin‐based chemotherapy in patients…
Continuous hemoperfusion, a technique used to filter toxins from blood, was effective in improving the survival rates of people with acute poisoning due to paraquat, an herbicide used in lawn care. The technique may have implications for the treatment of patients with paraquat-induced pulmonary fibrosis (PF), a…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…
