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Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases

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posted on 2018-02-28, 09:48 authored by Y. Schuller, C. E. M. Hollak, C. C. Gispen-de Wied, V. Stoyanova-Beninska, M. Biegstraaten
The full text of this article can be found here: https://link.springer.com/article/10.1007/s40265-017-0788-z

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    orphan medicinal productsmetabolic diseasesreal-world effectiveness

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