Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…
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A compound called tubastatin inhibits an enzyme-linked cell signaling pathway associated with lung tissue scarring, reducing pulmonary fibrosis, a study reports. The research on the HDAC6 enzyme in laboratory cell cultures and mice was able to identify only part of the mechanism involved in alleviating the scarring, however. The full…
Inhibiting an enzyme called phosphoglycerate dehydrogenase prevented additional lung tissue scarring in a mouse model of pulmonary fibrosis, a study reports. The discovery means scientists might be able to develop a PF therapy by targeting the enzyme, which is involved in metabolism, or transforming food into energy. The enzyme is…
The U.S. Food and Drug Administration has granted Fast Track designation to PBI-4050, ProMetic Life Sciences’ potential treatment for idiopathic pulmonary fibrosis, or IPF. Regulators recently approved ProMetic’s Investigational New Drug application for PBI-4050 and the company’s design for pivotal Phase 2/3 clinical trials of the therapy. A pivotal…
Ofev (nintedanib) is effective at slowing idiopathic pulmonary fibrosis (IPF) progression in patients over time regardless of their degree of lung function at the beginning of treatment, according to new results from an extension of a Phase 3 clinical trial. Ofev, by Boehringer Ingelheim, was approved by the U.S. Food and Drug…
Researchers have identified a variation of the AKAP13 gene that they say increases people’s susceptibility to developing idiopathic pulmonary fibrosis. An article about their discovery, published in The Lancet Respiratory Medicine, is titled “Genetic variants associated with susceptibility to idiopathic pulmonary fibrosis in people of European ancestry: a genome-wide association study.” Previous…
Global Blood Therapeutics (GBT), a clinical-stage biopharma focused on developing novel therapeutics for blood-based disorders, is discontinuing its GBT440 program for the treatment of idiopathic pulmonary fibrosis (IPF). The decision came after GBT evaluated the results from three studies, including one Phase 1 study and two Phase 2a…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
The diabetes therapy vildagliptin reduces lung scarring after bacteria-related lung damage, according to a study in mice. It does this by preventing endothelial cells that line lung blood vessels from transforming themselves into stem cells — a process lungs use to protect themselves from the bacteria-related attack, known as an…
Results from the RECAP clinical trial, which was conducted over five years, support the long-term safety of Esbriet (pirfenidone) in patients with idiopathic pulmonary fibrosis (IPF). The study, “An Open-Label Study of the Long-Term Safety of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis (RECAP),” was recently published in…
