The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…
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The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
Investigational SM04646 inhibits expression of TGF-beta-stimulated genes known to promote lung fibrosis, a new study shows. The results will be presented in a poster titled “SM04646 Inhibited Wnt Pathway Gene Expression Stimulated in Response to Transforming Growth Factor-beta and was Effective in a Chronic Model of Bleomycin-induced Pulmonary…
Two molecules secreted by epithelial cells of the lungs — CST3 and GDF15 — “are bona fide regulators” of scarring processes and may point to a new way to treat pulmonary fibrosis, an early study reports. The study, “Epithelial cell-derived cytokines CST3 and GDF15 as potential therapeutics…
Treatment with Esbriet (pirfenidone, Genentech) or Ofev (nintedanib, Boehringer Ingelheim), or with steroids was found to be safe for idiopathic pulmonary fibrosis (IPF) patients needing a bilateral lung transplant (BLTx), a study reports. The research, “Effect of Antifibrotics on Short-Term Outcome after Bilateral Lung…
The FOXM1 gene is a driver of lung fibroblast proliferation and activation in idiopathic pulmonary fibrosis (IPF), a new study shows. The findings highlight the therapeutic potential of targeting FOXM1 for pulmonary fibrosis treatment. The study “FOXM1 is a critical driver of lung fibroblast activation and fibrogenesis” was published in…
Like many of those living with serious chronic diseases such as idiopathic pulmonary fibrosis (IPF), Marj Korn depends on nurses to help her through the difficult journey. “They have always been kind and made sure to make me as comfortable as possible,” said the former senior recruitment manager. “I need…
The University of Virginia recently launched the Fibrosis Initiative, a platform intended to support and mobilize researchers to collaborate and conduct studies focused on fibrosis. This program is expected to expand knowledge on fibrosis and lead to potentially groundbreaking discoveries that could open up new therapeutic opportunities. “In many…
Treatment with regulatory T-cells (Tregs), a subset of cells from the immune system, is able to resolve lung fibrosis in a mouse model of idiopathic pulmonary fibrosis (IPF), according to researchers. Their findings support additional investigation of cell-based therapies to treat IPF in…
At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…
