Company News

Marketing Authorization Application for KIZFIZO® accepted for review by the EMA for the treatment of malignant glioma London, UK, March 30, 2026 – Orphalan, a pharmaceutical company dedicated to the…

On #InternationalRareDiseaseDay 2026, we join the global movement to recognise 300 million people worldwide living with a rare disease. At Orphalan, our mission is clear: to develop and deliver treatments for…

Over the years, I’ve been deeply moved by the stories of many patients, but one particularly stands out. A woman talked about her teenage daughter’s Wilson disease — a rare…

Why We Should Care About Rare Disease Research “Access to life-saving or life-enhancing treatments should not be determined by the rarity of the disease; on the contrary, our greater understanding…

DNA Today is a multi-award-winning podcast and radio show exploring how genetics impacts our health. Host/Producer Kira Dineen interviews leaders in genetics including genetic counsellors, researchers, physicians, and patient advocates…

Friday 25 June Today we are excited to announce positive top-line data from our Phase 3 head-to-head trial of trientine tetrahydrochloride and d-penicillamine. This is the first trial of its…