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…
- Cuvrior™ now available in the US for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to penicillamine - Oral administration offers dosing flexibility…
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…
Dr. Naseem Amin, Chief Executive Officer at Orphalan, discusses the announcement of the FDA approval of Cuvrior™ in this video: https://www.youtube.com/watch?v=Yu2WsUwtayE
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…