The discovery of medicines for rare diseases

Validating therapeutic targets through human genetics

(2013) Robert M. Plenge et al.   NATURE REVIEWS DRUG DISCOVERY

Vx-770 potentiates CFTR function by promoting decoupling between the gating cycle and ATP hydrolysis cycle

(2013) Kang-Yang Jih et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Physiology and Pharmacology of Plerixafor

(2013) Simon P. Fricker   TRANSFUSION MEDICINE AND HEMOTHERAPY

Ruxolitinib

(2012) Ruben A. Mesa et al.   NATURE REVIEWS DRUG DISCOVERY

Vemurafenib: the first drug approved for BRAF-mutant cancer

(2012) Gideon Bollag et al.   NATURE REVIEWS DRUG DISCOVERY

Ivacaftor

(2012) Pamela B. Davis et al.   NATURE REVIEWS DRUG DISCOVERY

Rare diseases and orphan drugs

(2012) Irena Melnikova   NATURE REVIEWS DRUG DISCOVERY

Enhancing Neural Transmission in Multiple Sclerosis (4-Aminopyridine Therapy)

(2012) Andrew D. Goodman et al.   Neurotherapeutics

Ponatinib in Refractory Philadelphia Chromosome–Positive Leukemias

(2012) Jorge E. Cortes et al.   NEW ENGLAND JOURNAL OF MEDICINE

Glucarpidase to combat toxic levels of methotrexate in patients

(2012) Jacalyn Green   Therapeutics and Clinical Risk Management

Omacetaxine as an Anticancer Therapeutic: What is Old is New Again

(2011) Meir Wetzler et al.   CURRENT PHARMACEUTICAL DESIGN

Clinical pharmacology as a cornerstone of orphan drug development

(2011) Edward D. Bashaw et al.   NATURE REVIEWS DRUG DISCOVERY

How were new medicines discovered?

(2011) David C. Swinney et al.   NATURE REVIEWS DRUG DISCOVERY

Crizotinib

(2011) Alice T. Shaw et al.   NATURE REVIEWS DRUG DISCOVERY

Role of carglumic acid in the treatment of acute hyperammonemia due to N-acetylglutamate synthase deficiency

(2011) Johannes Häberle   Therapeutics and Clinical Risk Management

Structural Mechanism of the Pan-BCR-ABL Inhibitor Ponatinib (AP24534): Lessons for Overcoming Kinase Inhibitor Resistance

(2010) Tianjun Zhou et al.   Chemical Biology & Drug Design

The Role of Heme and the Mitochondrion in the Chemical and Molecular Mechanisms of Mammalian Cell Death Induced by the Artemisinin Antimalarials

(2010) Amy E. Mercer et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Discovery of Artemisinin (Qinghaosu)

(2009) Fulong Liao   MOLECULES

Raxibacumab for the Treatment of Inhalational Anthrax

(2009) Thi-Sau Migone et al.   NEW ENGLAND JOURNAL OF MEDICINE