Rare diseases

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

Views & Analysis

Orphan drugs will fuel pharma's future - and a pricing pushb...

Evaluate predicts sales could approach $8bn if approved.

FDA fast tracks Shire clotting disease drug

Horses or zebras: inspiring behavioural change to make the r...

Thanks Mr President, but you’re wrong about FDA, says rare d...

Teva files orphan blockbuster hope in tardive dyskinesia

Kite’s CAR-T therapy most valuable pipeline orphan drug

Orphan drugs will fuel pharma's future - and a pricing pushb...

GBL joins CVC in €10.7bn takeover bid for Recordati

Discover a new era of healthcare panel engagement: Grounded ...

Rare diseases

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