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...

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Pubblicato in:J Biopharm Stat
Autori principali: Basu, Cynthia, Ahmed, Mariam A., Kartha, Reena V., Brundage, Richard C., Raymond, Gerald V., Cloyd, James C., Carlin, Bradley P.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2016
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Article
Accesso 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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