How Often are Orphan Drugs Orphaned by the Thermochemical Community?

Author(s): Kathleen F. Edwards*, Joel F. Liebman*

Journal Name: Current Medicinal Chemistry

Volume 27 , Issue 1 , 2020

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Orphan drug products (e.g. drugs and biologics) in the United States are those that treat people with rare chronic diseases, often cancer or metabolic disease. The rare disease condition being treated by these orphan drugs must serve a patient population of less than 200,000 people in the U.S. in order to earn the orphan drug product title. Just as the disease conditions are seen as “orphans,” so, we assert is the thermochemical understanding of the drugs themselves in terms of the chemical structures that define those drugs. This article illustrates this orphan thermochemical status for a recent series of orphan drugs.

Keywords: Orphan drug products, thermochemical, orfadin, fabrazyme, metabolic disease, thermo-chemical drugs.

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Year: 2020
Published on: 18 February, 2020
Page: [23 - 31]
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DOI: 10.2174/0929867325666181101120058
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