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Development of a model‐based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy
Early clinical trials of therapies to treat Duchenne muscular dystrophy (DMD), a fatal genetic X‐linked pediatric disease, have been designed based on the limited understanding of natural disease progression and variability in clinical measures over different stages of the continuum of the disease....
| Autores principales: | , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8923721/ https://www.ncbi.nlm.nih.gov/pubmed/34877803 http://dx.doi.org/10.1002/psp4.12753 |