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...

Full description

Saved in:
Bibliographic Details
Published in:J Biopharm Stat
Main Authors: Basu, Cynthia, Ahmed, Mariam A., Kartha, Reena V., Brundage, Richard C., Raymond, Gerald V., Cloyd, James C., Carlin, Bradley P.
Format: Artigo
Language:Inglês
Published: 2016
Subjects:
Article
Online Access: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
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items