Repurposing as a strategy for orphan drug development, evidence from European approvals

Orphan drug approvals in Europe since 2001: an analysis by indication

(2013) Peter Norman   Expert Opinion on Orphan Drugs

Global Phenotypic Screening for Antimalarials

(2012) W. Armand Guiguemde et al.   CHEMISTRY & BIOLOGY

A generalizable pre-clinical research approach for orphan disease therapy

(2012) Chandree L Beaulieu et al.   Orphanet Journal of Rare Diseases

Big Pharma's new model in orphan drugs and rare diseases

(2012) M Ian Phillips   Expert Opinion on Orphan Drugs

Randomized placebo-controlled trial of the bradykinin B2 receptor antagonist icatibant for the treatment of acute attacks of hereditary angioedema: the FAST-3 trial

(2011) William R. Lumry et al.   ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY

In silico repositioning of approved drugs for rare and neglected diseases

(2011) Sean Ekins et al.   DRUG DISCOVERY TODAY

Sustained remission of symptoms and improved health-related quality of life in patients with cryopyrin-associated periodic syndrome treated with canakinumab: results of a double-blind placebo-controlled randomized withdrawal study

(2011) Isabelle Koné-Paut et al.   ARTHRITIS RESEARCH & THERAPY

The bradykinin-forming cascade and its role in hereditary angioedema

(2010) Allen P. Kaplan et al.   ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY