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Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease
Approximately 50% of rare diseases are evident in children. Fatal disease prognosis and lack of treatments causes 30% of affected children to not live past their fifth birthday. This clear sense of urgency demands innovation and acceleration in drug development. A case study is discussed highlightin...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2016
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5102572/ https://www.ncbi.nlm.nih.gov/pubmed/27351288 http://dx.doi.org/10.1002/cpt.417 |