Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy

Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy

Abstract Background Centronuclear myopathies are severe rare congenital diseases. The clinical variability and genetic heterogeneity of these myopathies result in major challenges in clinical trial design. Alternative strategies to large placebo-controlled trials that have been used in other rare di...

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Bibliographic Details
Main Authors: Eve Fouarge, Arnaud Monseur, Bruno Boulanger, Mélanie Annoussamy, Andreea M. Seferian, Silvana De Lucia, Charlotte Lilien, Leen Thielemans, Khazal Paradis, Belinda S. Cowling, Chris Freitag, Bradley P. Carlin, Laurent Servais, the NatHis-MTM Study Group
Format: Article
Language:English
Published: BMC 2021-01-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Bayesian analysis
Centronuclear myopathy
Complex innovative clinical trial design
Disease progression model
Natural history data
Online Access:https://doi.org/10.1186/s13023-020-01663-7

Internet

https://doi.org/10.1186/s13023-020-01663-7

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