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Drugs for rare disorders.

Serge Cremers¹, Jeffrey K Aronson²

¹Departments of Pathology & Cell Biology and Medicine, and Irving Institute for Clinical and Translational Research, Columbia University Medical Center, New York, NY, 10027, USA.

British Journal of Clinical Pharmacology

|June 28, 2017

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Collectively, rare disorders affect millions globally, often impacting children and having genetic origins. Developing treatments, or orphan drugs, faces challenges, but clinical pharmacology plays a key role.

Area of Science:

Rare disease epidemiology
Genetics
Clinical pharmacology

Background:

Rare disorders collectively affect a significant portion of the population (6-8% in the US).
Most rare disorders are genetically determined and frequently impact children.
Diagnosis is often challenging due to variable presentations and limited clinician experience.

Keywords:

clinical pharmacology drug development orphan drugs orphan products rare diseases rare disorders