More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…
Quick Approval Given COVID Vaccines Raise Concerns, Rare Disease Patients Say
A variant of the MUC5B gene, known as the rs35705950 (T) allele, appears to lead to better survival in people with idiopathic pulmonary fibrosis (IPF) on anti-fibrotic treatment, a study reports. These findings highlight the potential usefulness of MUC5B genetic data in treating and managing these patients. The study,…
United Therapeutics has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) requesting the approval of Tyvaso DPI, an investigational dry powder inhaled formulation of treprostinil, to treat pulmonary hypertension associated with…
Outcomes are as poor in people with progressive fibrosing interstitial lung disease (PF-ILD) other than idiopathic pulmonary fibrosis (IPF) as in those with IPF, according to a Japanese real-world study. Researchers also found that worse lung function at a first assessment and the presence of fibrotic patterns in the…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…
Often used in traditional Chinese medicine, a plant compound called salvianolic acid B, or SAB, has been found to reduce lung scarring (fibrosis) and blood clotting when given in an inhaled form to a rat model of idiopathic pulmonary fibrosis (IPF). These findings suggest that SAB should be…
A study published early last year revealed that patients living with a rare disease have a greater risk of developing anxiety and other mental health issues compared with people with more common diseases, The Lund Report noted. While more than 6,000 rare diseases have been identified around the…
A patient with idiopathic pulmonary fibrosis (IPF) who also had non-small cell lung cancer responded well to treatment with Ofev (nintedanib), according to a new case report. The findings suggest Ofev might be an effective treatment choice for patients in whom the two conditions coexist but who are…
Scientists at the University of Nottingham, in the U.K., are asking pulmonary fibrosis (PF) patients — many of whom donate “gifts” of cells and samples — and their caregivers to help define the institution’s future research goals. These researchers, from the university’s faculty of medicine and health sciences and…
Axatilimab, an investigational therapy being developed by Syndax Pharmaceuticals, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) as a potential treatment for idiopathic pulmonary fibrosis (IPF). The FDA gives orphan drug status to medications that have the potential to substantially improve care…
