News

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

A new subtype of stem cells that have the unique ability to perform two distinct functions at the same time was recently discovered by researchers at University of Queensland (UQ) in Australia. These cells can generate both endothelial and mesenchymal cells, meaning they hold the potential to regenerate or…

In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…

A Phase 3 clinical trial in idiopathic pulmonary fibrosis (IPF) patients will be a top clinical development priority for Prometic Life Sciences in 2018, the company announced. The realignment of the company’s research priorities follows a meeting held with the U.S. Food and Drug Administration (FDA) in January 2018.

PMD Healthcare is creating advisory boards composed of pulmonary fibrosis and cystic fibrosis patients and caregivers. The company, whose services include remote monitoring of patients’ health conditions, is in the midst of recruiting members of both boards. PMD said the purpose of the boards is to gather information and opinions directly…

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…

PBI-4050, Prometic Life Sciences’ lead therapy candidate, works through a dual mechanism to target a newly discovered pathway, involving the receptors GPR40 and GPR84, that is critical to fibrosis development, a study shows. The study, “A Newly Discovered Antifibrotic Pathway Regulated by Two Fatty Acid Receptors:…

An enzyme can start a chain of events that leads to cells involved in tissue scarring reversing their fibrosis-generating activity, British and American researchers have discovered. The study dealt with the enzyme’s effect on myofibroblasts — cells involved in wound healing and inflammatory response. The discovery could lead to new therapies for the lung scarring…

Kadmon’s KD025 slowed the progression of idiopathic pulmonary fibrosis and patients’ decline in lung function, a Phase 2 clinical trial shows. The IPF patients in the open-label trial (NCT02688647) had previously been treated with Genentech’s Esbriet (pirfenidone) or Boehringer Ingelheim’s  Ofev (nintedanib) — or had…

patientMpower, a U.K. digitial healthcare and advocacy company, joined others in voicing disappointment with the recent decision of a British advisory board to restrict the use of Esbriet (pirfenidone) to patients with moderate-to-severe idiopathic pulmonary fibrosis (IPF). NICE’s final recommendations for Esbriet (marketed by Genentech) and its…