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A Hierarchical Bayesian Approach for Combining Pharmacokinetic/Pharmacodynamic Modeling and Phase IIa Trial Design in Orphan Drugs: Treating Adrenoleukodystrophy with Lorenzo’s Oil
X-linked adrenoleukodystrophy (X-ALD) is a rare, progressive and typically fatal neurodegenerative disease. Lorenzo’s Oil (LO) is one of the few X-ALD treatments available, but little has been done to establish its clinical efficacy or indications for its use. In this paper, we analyze data on 116 m...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | J Biopharm Stat |
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| Κύριοι συγγραφείς: | , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
2016
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5393457/ https://ncbi.nlm.nih.gov/pubmed/27547896 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/10543406.2016.1226326 |
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