While in 2021, orphan drugs made up over half of all the US FDA’s approvals, with the figures in European being similarly high – 19 out of 54 approved new active substances.

Neil Dickinson continues his discussion around rare diseases – this time, by looking at the marketing of orphan drugs by pharmaceutical companies. (Continued from “Rare disease part 1 ...

EMA grants orphan drug designation to Quoin Pharma’s QRX003 for the treatment of Netherton syndrome: Ashburn, Virginia Thursday, May 22, 2025, 15:00 Hrs [IST] Quoin Pharmaceutic ...

The Orphan franchise, known for its chilling psychological horror, is potentially poised for a third installment following the surprising success of its 2022 prequel, Orphan: First Kill.

Delhi, May 14, 2025 (GLOBE NEWSWIRE) -- US Orphan Designated Drugs Market Opportunity, Drugs Sales, Price, Dosage and Clinical Trials Insight 2030 Report Offering and Highlights: This report on ...

The European Medicines Agency (EMA) has designated allopurinol as the first orphan drug for the treatment of Marfan syndrome, a rare connective tissue disease which has no cure to date.

It may have been hard to predict back in 2009, but Isabelle Fuhrman's Esther has become something of a horror movie mainstay thanks to the film Orphan and its 2022 prequel, Orphan: First Kill.

A phase 2 trial (ClinicalTrials.gov Identifier ... Data from the study are expected in 2026. “Receiving Orphan Drug designation for TPST-1495, our second clinical program, is a significant ...