How many #orphandrugs have marketing authorization in Europe? How many drugs without orphan status are used in rare diseases? All these questions and more answered in the new Orphanet Report Series on #raredisease drugs in Europe: https://buff.ly/3rUBw24 | Orphanet | Facebook

It’s that time of the year again, and our team is thrilled to be returning to the World Orphan Drugs Congress USA 2024 in Boston! Rachel Smith, Head of Rare Disease at Parexel, leads an esteemed panel, including Peter Marks from the FDA, Mark Trusheim from Tufts Medical Center, Jamie Sullivan from EveryLife Foundation for Rare Diseases, and Kinnari Patel from Rocket Pharmaceuticals, exploring the latest regulatory challenges and opportunities in accelerating rare disease treatments for patients