News Charities unite to oppose NICE rare disease rules MPs to discuss controversial plan in special debate.
News FDA fast tracks Shire clotting disease drug The US regulator has given a Fast Track designation to a Shire pipeline drug for a life-threatening blood clotting disease, hereditary thrombotic thrombocytopenic purpura.
Views & Analysis Horses or zebras: inspiring behavioural change to make the r... Insight into human behaviour allows development of programmes that help doctors diagnose rare diseases.
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
News UK's Mission divests kidney drug to Dimerix for $292m Mission Therapeutics has struck a deal to out-license a drug for acute kidney injury to Australia's Dimerix, sharpening its focus on CNS diseases.
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