News Thanks Mr President, but you’re wrong about FDA, says rare d... NORD patient advocacy group responds to Congress speech
News Kite’s CAR-T therapy most valuable pipeline orphan drug Drug tops list compiled by Evaluate Pharma
Views & Analysis Orphan drugs will fuel pharma's future - and a pricing pushb... Evaluate predicts sales could approach $8bn if approved.
Views & Analysis Rare diseases and orphan drugs need digital innovation mHealth tech can provide longer-term monitoring for clinical trials.
News Biogen/Ionis could launch rare disease drug within weeks Companies prepare for launch of Spinraza after phase 3 success.
Views & Analysis Are European incentives speeding access in rare cancer treat... Examining the impact of support programmes for orphan treatments.
