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...

Cur síos iomlán

Na minha lista:
Sonraí Bibleagrafaíochta
Foilsithe 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.
Formáid: Artigo
Teanga:Inglês
Foilsithe: 2016
Ábhair:
Article
Rochtain Ar Líne: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
Clibeanna: Cuir Clib Leis
Gan Chlibeanna, Bí ar an gcéad duine leis an taifead seo a chlibeáil!
Á lódáil...

Míreanna Comhchosúla