Therapeutic Orphan No More: Role for Clinical Pharmacology and Translational Science in Developing Therapeutics for Rare and Neglected Diseases

Affiliations

• ¹ Potomac, Maryland, USA.
• ² Quantitative Pharmacology and Pharmacometrics, Merck and Co. Inc, Rahway, New Jersey, USA.
• ³ Gilead Sciences Inc., Foster City, California, USA.
• ⁴ Division of Applied Regulatory Science, Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
• ⁵ Department of Bioengineering and Therapeutic Sciences, University of California, San Francisco, California, USA.
• ⁶ Certara, Applied BioSimulation, Canterbury, UK.
• ⁷ Leiden Academic Centre for Drug Research, Leiden, The Netherlands.