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Novel clinical trial design and analytic methods to tackle challenges in therapeutic development in rare diseases
While only a fraction of the worldwide population may have a particular rare disorder, millions of people worldwide are affected across the over 6,000 rare disorders and do not have a safe and effective approved therapy to help them live or manage complications from the disorder. Challenges to clini...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
AME Publishing Company
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9577738/ https://www.ncbi.nlm.nih.gov/pubmed/36267797 http://dx.doi.org/10.21037/atm-21-5496 |