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 Teva files orphan blockbuster hope in tardive dyskinesia SD-809 will be vital for Teva if approved.
News Kite’s CAR-T therapy most valuable pipeline orphan drug Drug tops list compiled by Evaluate Pharma
News Adcendo raises $135m for cancer ADCs, and other financings Adcendo raises $135m for cancer ADCs, plus financings for Enveda, Cradle Bio, ATB Therapeutics, and 35Pharma
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