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Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases
The literature thoroughly describes the challenges of pediatric drug development for rare diseases. This includes (1) generating interest from sponsors, (2) small numbers of children affected by a particular disease, (3) difficulties with study design, (4) lack of definitive outcome measures and ass...
Autores principales: | Epps, Carla, Bax, Ralph, Croker, Alysha, Green, Dionna, Gropman, Andrea, Klein, Agnes V., Landry, Hannah, Pariser, Anne, Rosenman, Marc, Sakiyama, Michiyo, Sato, Junko, Sen, Kuntal, Stone, Monique, Takeuchi, Fumi, Davis, Jonathan M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer International Publishing
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9040360/ https://www.ncbi.nlm.nih.gov/pubmed/35471559 http://dx.doi.org/10.1007/s43441-022-00409-w |
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