Dynamic enrichment of Bayesian small-sample, sequential, multiple assignment randomized trial design using natural history data: a case study from Duchenne muscular dystrophy.

Wang, Sidi; Kidwell, Kelley M; Roychoudhury, Satrajit

Wang, Sidi; Kidwell, Kelley M; Roychoudhury, Satrajit.

Afiliación

Wang S; Department of Biostatistics, University of Michigan, Ann Arbor, Michigan, USA.
Kidwell KM; Department of Biostatistics, University of Michigan, Ann Arbor, Michigan, USA.
Roychoudhury S; Pfizer Inc., New York, New York, USA.

Biometrics ; 79(4): 3612-3623, 2023 12.

Article en En | MEDLINE | ID: mdl-37323055

Asunto(s)

Distrofia Muscular de Duchenne; Humanos; Distrofia Muscular de Duchenne/tratamiento farmacológico; Teorema de Bayes; Enfermedades Raras

Palabras clave

Bayesian hierarchical model; Duchenne muscular dystrophy; clinical trial; rare disease; small sample

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Distrofia Muscular de Duchenne Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Humans Idioma: En Revista: Biometrics Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos

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