Volume 38, Issue 1
  • ISSN: 0002-9637
  • E-ISSN: 1476-1645


This colloquium held in September 1985 presents an interesting survey of the question “Can the problem of orphan diseases and orphan drugs be directly attacked?”

The first third of the book is on Wilson's disease and the drugs d-Penicillamine and trientine. The papers are technical and represent the original nucleus of the sponsor's interest in establishing a Fifth International Congress just on Wilson's disease. The reports do serve to place in perspective the technical issues facing all drugs and diseases including orphan drugs and orphan diseases.

The last two thirds of the symposium are directly related to general aspects of orphan drugs and diseases. Congressman Henry Waxman, the sponsor of the U.S. Orphan Drug Act, and his British counterpart, R. D. Mann, surveyed the history of the regulatory requirements in the two countries. Waxman outlined the amendments of 1984 and 1985 establishing the National Commission of Orphan Diseases and extending support to preclinical areas.


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