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How do we approach the use of nonapproved therapy in treating rare conditions? Beneficence (literally "doing good") must be the first concern and it relates to the fundamental requirement (Hippocratic Oath) to do no harm. Certainly, it would be harmful to the patient to avoid potentially efficacious treatments because the demographics of evidence-based medicine are below the threshold for regulatory approval.
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This page is a summary of: Rational therapy: A defense against evidence-based medicine, Movement Disorders, March 2011, Wiley,
DOI: 10.1002/mds.23454.
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