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

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 diseases (...

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Detalles Bibliográficos
Autores principales: Fouarge, Eve, Monseur, Arnaud, Boulanger, Bruno, Annoussamy, Mélanie, Seferian, Andreea M., De Lucia, Silvana, Lilien, Charlotte, Thielemans, Leen, Paradis, Khazal, Cowling, Belinda S., Freitag, Chris, Carlin, Bradley P., Servais, Laurent
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7789189/
https://www.ncbi.nlm.nih.gov/pubmed/33407688
http://dx.doi.org/10.1186/s13023-020-01663-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7789189/
https://www.ncbi.nlm.nih.gov/pubmed/33407688
http://dx.doi.org/10.1186/s13023-020-01663-7

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