Rare Disease Report


Researchers find that a specially formulated antioxidant-enriched supplement may decrease respiratory illnesses in patients with cystic fibrosis.
Study show blocking FOXM1 can reduce the activation of fibroblasts as well as the process of idiopathic pulmonary fibrosis (IPF) itself.
Positive pre-clinical data is released regarding BGB601 for the treatment of fibrosis, idiopathic pulmonary fibrosis (IPF), and non-alcoholic steatohepatitis (NASH).
At the most recent American Lung Association LUNGFORCE expo, RDR sat down with Albert Rizzo, M.D. of the Christiana Care Pulmonary and Critical Care Medicine Section to discuss previous, current and emerging therapies for idiopathic pulmonary fibrosis.
Clinical data from an ongoing Phase 2 study of PBI-4050 shows that the drug provides a clear clinical benefit in patients with Alstrom syndrome.
The American Journal of Pathology  published a paper detailing the activity of the mechanism of action PBI-4050 in IPF.
Arrowhead Pharmaceuticals Inc. announced that the company’s second-generation investigational medicine ARO-AAT was granted orphan drug designation by the FDA.
Faron Pharmaceuticals announced this morning that the U.S. FDA has approved the Investigational New Drug application for Traumakine (FP-1201-lyo) for the treatment of Acute Respiratory Distress Syndrome.
Rare Disease Report spoke with pulmonologist Dr. Marlies S. Wijsenbeek, Department of Respiratory Medicine, Erasmus University Medical Center (Rotterdam The Netherlands) about newly-published data in IPF.
Aradigm Corporation’s meeting with the Antimicrobial Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) is slated for January 11, 2018.
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