Development and Use of Gene Therapy Orphan Drugs—Ethical Needs for a Broader Cooperation Between the Pharmaceutical Industry and Society

Development and Use of Gene Therapy Orphan Drugs—Ethical Needs for a Broader Cooperation Between the Pharmaceutical Industry and Society

Gene therapy orphan medicinal products constitute a unique group of new drugs which in case of hereditary diseases are usually administered only once at an early age, in the hope to provide sufficient gene product to last for the entire life of the patients. The combination of an exceptionally large...

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Bibliographic Details
Main Authors: Sandor Kerpel-Fronius, Varvara Baroutsou, Sander Becker, Roberto Carlesi, Luis Collia, Brigitte Franke-Bray, Peter Kleist, Chieko Kurihara, Luis Filipe Laranjeira, Kotone Matsuyama, Shehla Naseem, Johanna Schenk, Honorio Silva
Format: Article
Language:English
Published: Frontiers Media S.A. 2020-12-01
Series:Frontiers in Medicine
Subjects:
gene therapy
rare diseases
orphan drugs
ethics
accelerated approval
health care
Online Access:https://www.frontiersin.org/articles/10.3389/fmed.2020.608249/full

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